AzTrials
AzTrials

Thrombotic Thrombocytopenic Purpura (TTP)Phase 3NCT03393975

A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)

A Phase 3 study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Baxalta now part of Shire at 33 sites in 9 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop. Read more about this study

Who is it for?
Ages 0 to 70 years
What is being tested?
TAK-755
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
52 participants
Where
33 sitesin 9 countries
Study dates
13 Oct 2017to 30 May 2024
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participant or legally authorized representative has provided signed informed consent >= 18 years of age and/or assent form (signed by legal representative if participants is <18 years of age).
  • Participant is 0 to 70 years of age, inclusive, at the time of screening. (Participants < 18 years of age will be enrolled only after at least 5 adults (>= 18 years of age) each have at least 10 exposures with BAX 930 and reviewed by the Data Monitoring Committee (DMC). In France, no participants younger than 18 years of age will be enrolled into the study before the first adult participant has been treated with BAX 930 for a minimum of 6 months.
  • Participant has a documented diagnosis of severe hereditary ADAMTS13 deficiency, defined as:
  • Confirmed by molecular genetic testing, documented in participant history or at screening, and
  • ADAMTS13 activity < 10 % as measured by the fluorescent resonance energy transfer- von Willebrand factor73 (FRETS-VWF73) assay, documented in participant history or at screening (participants currently receiving standard of care (SoC) prophylactic therapy may exceed 10% ADAMTS13 activity at screening).
  • Note: Participants currently receiving prophylactic therapy will be screened immediately prior to their usual prophylactic infusion
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  • Participant does not display any severe thrombotic thrombocytopenic purpura (TTP) signs (platelet count < 100,000/ microliter (mcL) and elevation of lactate dehydrogenase (LDH) greater than (>2)* ULN) at screening. (Prophylactic cohort only).
  • Participant is currently on a prophylactic dosing regimen or has a documented history of at least 1 TTP event and an ability to tolerate SoC prophylactic dosing (prophylactic cohort only).
  • Participants >= 16 years of age must have a Karnofsky score >= 70% and participants < 16 years of age must have a Lansky score >= 80%.
  • Participant is hepatitis C virus (HCV)-negative as confirmed by antibody or polymerase chain reaction testing OR HCV-positive if their disease is chronic but stable.
  • If female of childbearing potential, participant presents with a negative blood or urine pregnancy test, confirmed no more than 7 days before the first administration, and agrees to employ adequate birth control measures for the duration of the study and to undergo quarterly pregnancy testing.
  • Sexually active males must use an accepted and effective method of contraception during the treatment and until a minimum of 16 days after the last dose administered.
  • Participant is willing and able to comply with the requirements of the protocol.

Who cannot join

  • Participant has been diagnosed with any other TTP-like disorder (microangiopathic hemolytic anemia), including acquired TTP.
  • Participant has known hypersensitivity to hamster proteins.
  • Participant has experienced an acute TTP event less than 30 days prior to screening (prophylactic cohort only).
  • Participant has a medical history or presence of a functional ADAMTS13 inhibitor at screening.
  • Participant has a medical history of genetic or acquired immune deficiency that would interfere with the assessment of product immunogenicity, including participants who are human immunodeficiency virus (HIV)-positive with an absolute cluster of differentiation 4 (CD4) count < 200/ cubic millimeter (mm^3) or who are receiving chronic immunosuppressive drugs.
  • Participant has been diagnosed with severe cardiovascular disease (New York Heart Association classes 3 to 4).
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  • Participant with end stage renal disease requiring chronic dialysis.
  • Participant has been diagnosed with hepatic dysfunction, as evidenced by, but not limited to, any of the following:
  • Serum alanine aminotransferase (ALT) >= 2* ULN.
  • Severe hypoalbuminemia < 24 gram per liter (g/L).
  • Portal vein hypertension (e.g., presence of otherwise unexplained splenomegaly, history of esophageal varices).
  • In the opinion of the investigator, the participant has another clinically significant concomitant disease that may pose additional risks for the participant.
  • Participant has been treated with an immunomodulatory drug, excluding topical treatment (e.g., ointments, nasal sprays), within 30 days prior to enrollment. Use of corticosteroids in conjunction with administration of fresh frozen plasma (FFP) to prevent allergic reactions is permitted.
  • Participant has an acute illness (e.g., influenza, flu-like syndrome, allergic rhinitis/conjunctivitis, bronchial asthma) at the time of screening (prophylaxis cohort only).
  • Participant is receiving or anticipates receiving another investigational drug and/or interventional drug within 30 days before enrollment.
  • Participant has a history of drug and/or alcohol abuse within the last 2 years.
  • Participant has a progressive fatal disease and/or life expectancy of less than 3 months.
  • Participant is identified by the investigator as being unable or unwilling to cooperate with study procedures.
  • Participant suffers from a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
  • Participant is a family member or employee of the sponsor or investigator.
  • If female, participant is pregnant or lactating at the time of enrollment.
  • Any contraindication to SoC medicinal product(s) as per local prescribing information.

Trial locations and contacts

33 sites in 9 countries.

33 sites in 9 countries

United States7 sites

  • Winship Cancer Institute

    Atlanta, Georgia, 30322

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Ohio State Univ College Of Medicine

    Columbus, Ohio, 43210

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • The Methodist Hospital

    Houston, Texas, 77030

  • Alliance for Childhood Diseases, Cure 4 the Kids Foundation

    Las Vegas, Nevada, 89135

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104

Other trials in United States

Spain6 sites

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, La Coruña, 15006

  • Hospital General Universitario de Alicante

    Alicante, 3010

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903

  • Hospital Universitario de Salamanca

    Salamanca, 37007

  • Hospital Universitario Virgen del Rocio

    Seville, 41013

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

Other trials in Spain

France5 sites

  • Hopital Claude Huriez - CHU Lille

    Lille, Nord, 59037

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015

  • Hôpital Saint-Antoine

    Paris, Paris, 75571

  • Hôpital Robert Debré - Paris

    Paris, 75019

  • CHU Saint Etienne - Hôpital Nord

    Saint-Priest-en-Jarez Cedex, Pays de la Loire Region, 42270

Other trials in France

Italy5 sites

  • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)

    Bergamo, 24127

  • Azienda Ospedaliera Universitaria "Policlinico - Vittorio Emanuele" (Presidio Ferrarotto Alessi)

    Catania, 90124

  • Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

    Milan, 20122

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    Roma, 168

  • Dipartimento di Medicina Traslazionale e di Precisione - "Sapienza" Universita di Roma

    Rome, 00161

Other trials in Italy

Japan3 sites

  • Medical Hospital, Tokyo Medical and Dental University

    Bunkyō City, Tokyo-To, 113-8519

  • Kyushu University Hospital

    Fukuoka, Fukuoka, 812-8582

  • Hyogo College of Medicine Hospital

    Nishinomiya-shi, Hyōgo, 663-8501

Other trials in Japan

Germany2 sites

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246

  • Universitaetsklinikum Jena

    Jena, Thuringia, 07747

Other trials in Germany

Poland2 sites

  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny

    Warsaw, 02-091

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776

Other trials in Poland

United Kingdom2 sites

  • University College London Hospitals

    London, Greater London, NW1 2PG

  • Royal Manchester Children's Hospital

    Manchester, M139WL

Other trials in United Kingdom

Austria1 site

  • AKH - Medizinische Universität Wien

    Vienna, 1090

Other trials in Austria

About this study

Official title: A Phase 3, Prospective, Randomized, Controlled, Open-label, Multicenter, 2 Period Crossover Study With a Single Arm Continuation Evaluating the Safety And Efficacy of BAX 930 (rADAMTS13) in the Prophylactic And On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP])

Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop. The increased clotting that occurs in TTP uses up the cells that help the blood to clot, called platelets. With fewer platelets available in the blood, bleeding problems can occur. People who have TTP may bleed underneath the skin forming purple bruises or purpura, or from the surface of the skin. TTP also can cause anemia, a condition in which red blood cells break apart faster than the body can replace them leading to lower than normal number of red blood cells.

A lack of activity in the ADAMTS13 enzyme, a protein in the blood involved in blood clotting, causes TTP. The enzyme breaks up another blood protein called von Willebrand factor that clumps together with platelets to form blood clots. Some people are born with this condition, others get the condition during their life. Many people who born with TTP experience frequent flareups that need to be treated right away. If not treated It can be fatal or cause lasting damage, such as brain damage or a stroke. BAX 930 is a medicine that replaces ADAMTS13 and can prevent or control TTP flareups, called TTP events.

Read more

The main aim of this study is to compare the number of TTP events in people born with severe TTP when they treated with BAX 930 versus when they are treated with the standard treatment. Treatment will be given in 2 ways:

  • BAX 930 or standard treatment given to prevent TTP events from happening.
  • BAX 930 or standard treatment given to control an acute TTP event when it happens, according to the clinic's standard practice.

Both BAX 930 and standard treatment are given slowly through a vein (infusion).

At the first visit, the study doctor will check if you can participate in the study. If you are eligible and enter the study, you will follow an assigned schedule and either start with BAX 930 (Period 1) and then switch to standard treatment (Period 2) or start with standard treatment (Period 1) and then switch to BAX 930 (Period 2). Everyone will be treated with BAX 930 again for Period 3. Each Period will last approximately 6 months.

If you enter the study to control an acute TTP event, you will follow a schedule receiving either BAX 930 or standard care to treat your acute TTP event. Once the acute TTP event has gotten better, you can decide to continue in the study and be given treatment to prevent TTP events from happening, following the schedule above.

Another study's aim is to assess side effects from treatment with BAX 930 and standard treatment. To do that, the study doctor will ask you questions about your health at each study visit.

The study doctors will also check how long BAX 930 stays in the blood of the participants, over time. They will do this from blood samples taken after participants receive their specific infusions of BAX 930. This will happen at different times during the study.

1 month after all treatment has been completed, participants will visit the clinic for a final check-up.

Being tested
TAK-755, Standard of care
Medical subject headings
Purpura, Thrombotic Thrombocytopenic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TAK-755Biological

    Also called rADAMTS13, SHP-655, recombinant ADAMTS13, BAX 930

    Participants in prophylaxis cohort will receive IV infusions of 40 IU/kg TAK-755 ORT during Period 1 and Period 2 and switch to TAK-755 SIN during Period 3 for six months once in a week or twice in a week. In On-demand cohort participants will receive daily IV dose of TAK-755.

  • Standard of careBiological

    Participants will receive Investigator-recommended Standard of care (SoC).

Study groups in NCT03393975
Study groupRoleWhat they receive
Prophylaxis Cohort I: TAK-755 Then SoCExperimentalParticipants received a single intravenous (IV) infusion of 40 international units per kilogram (IU/kg) rADAMTS13 manufactured in Orth, Austria (TAK-755 ORT), every 2 weeks (Q2W) for 6 months in Period 1 followed by standard of care (SoC) for 6 months in Period 2. Thereafter participants received rADAMTS13 manufactured in Singapore (TAK-755 SIN), dose IV infusion of 40 IU/kg Q2W for 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
Receives: Biological: TAK-755; Biological: Standard of care
Prophylaxis Cohort II: SoC Then TAK-755ExperimentalParticipants received SoC for 6 months in Period 1 followed by IV infusions of 40 IU/kg dose of TAK-755 ORT Q2W in Period 2 for the next 6 months. Thereafter participants received TAK-755 SIN dose IV infusions of 40 IU/kg Q2W for another 6 months in Period 3. TAK-755 ORT could be replaced with TAK-755 SIN and vice versa depending on availability and other criteria.
Receives: Biological: TAK-755; Biological: Standard of care
On Demand Cohort I: TAK-755ExperimentalParticipants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the TAK-755 cohort of the Urgent Treatment Period received initial dose of IV infusion 40 IU/kg [+/- 4 IU/kg] TAK-755 ORT or TAK-755 SIN on Day 1 followed by a subsequent dose IV infusions of 20 IU/kg [+/- 2 IU/kg] TAK-755 ORT or TAK-755 SIN on Day 2 and an additional daily dose IV infusions of 15 IU/kg [+/- 1.5 IU/kg] TAK-755 on Day 3 until 2 days after the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
Receives: Biological: TAK-755
On Demand Cohort II: SoCExperimentalParticipants experiencing an acute TTP event who met all other inclusion criteria and entered the study through the SoC cohort of the Urgent Treatment Period received the investigator-recommended SoC and dosing regimen until the acute event was resolved. Upon resolution of the acute TTP event, participants had the option to either move to the prophylaxis cohort of the study or discontinue entirely.
Receives: Biological: Standard of care

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment

    As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 74.5 months

Secondary outcomes (43)

  1. Percentage of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events Responding to TAK-755

    Percentage of acute TTP events responding to TAK-755, was defined as not requiring the use of another human disintegrin and metalloprotease with a thrombospondin type 1 motif, member 13 (ADAMTS13)-containing agent. As per planned analysis, data for this outcome measure were collected and reported only for the TAK-755 treatment arm of both the prophylaxis (irrespective of the prophylaxis periods) and on demand cohorts.

    Measured: Up to 79.6 months

  2. Time to Resolution of Acute TTP Events

    Time to resolution of acute TTP events following initiation of treatment with TAK-755 or SoC agent was assessed. Acute TPP events were considered resolved when: (a) Platelet count was >150,000 per microliter (μL) or drop of platelet count was within 25 percent (%) of baseline, whichever occurred first, and (b) Elevation of lactate dehydrogenase (LDH) <1.5 x baseline or <1.5 x upper limit of normal (ULN). As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the prophylaxis treatment Periods, partitioned per treatment received (TAK-755 and SoC) for the on demand and prophylactic cohorts.

    Measured: Up to 79.6 months

  3. Number of Participants With Thrombocytopenia During Prophylactic Treatment

    Thrombocytopenia was defined as a decrease in platelet count ≥25 % of baseline or a platelet count <150,000/μL, reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  4. Number of Participants With Microangiopathic Hemolytic Anemia During Prophylactic Treatment

    Microangiopathic hemolytic anemia was defined as an elevation of LDH >1.5* of baseline or >1.5*ULN (with a possible evidence of schistocytes on blood smear) and was reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  5. Number of Participants With Neurological Symptoms During Prophylactic Treatment

    Neurological symptoms (TTP related) (e.g., confusion, dysphonia, dysarthria, focal or general motor symptoms including seizures), were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  6. Number of Participants With Renal Dysfunction During Prophylactic Treatment

    Renal dysfunction was defined as an increase in serum creatinine >1.5*baseline. Number of participants with renal dysfunction were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  7. Number of Participants With Abdominal Pain During Prophylactic Treatment

    Number of participants with abdominal pain (TTP related) were reported by treatment arm for the prophylaxis cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  8. Number of Supplemental Doses Prompted by Subacute TTP Event During Prophylactic Treatment

    Number of supplemental doses prompted by subacute TTP events were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  9. Number of Participants With Dose Modification Not Prompted by an Acute TTP Event During Prophylactic Treatment

    Number of participants with dose modification not prompted by an acute TTP event were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  10. Number of Participants With Acute TTP Events on Their Final Dose

    Number of participants with acute TTP events on their final dose and dosing regimen for the prophylactic cohort were reported. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755.

    Measured: Up to 79.6 months

  11. Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Treatment Emergent Adverse Events (Serious TEAEs)

    AE: Any untoward medical occurrence in participants administered IP that does not necessarily have a causal relationship with treatment. TEAE: AE that has start date-time on/after start date-time of first dose of treatment participant is taking on that assessment/period or if it has start date-time before start date-time of first dose but increases in severity on/after start date-time of the first dose of treatment. SAE: An untoward medical occurrence that at any dose meets 1 or more of following criteria: death; initial/prolonged in-patient hospitalization; life threatening experience; persistent/significant disability/incapacity; congenital anomaly, medically important event (may not be immediately life threatening or result in death or require hospitalization but may require medical or surgical intervention to prevent 1 of the other outcomes). Vital signs, clinical chemistry, hematology as assessed by the investigator were reported as AE.

    Measured: Up to 79.6 months

  12. Number of Participants With Inhibitory Antibodies to ADAMTS13

    Number of participants with inhibitory antibodies to ADAMTS13 were reported. As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the Prophylaxis Periods and partitioned as per the treatment received during the course of the study, presented for the prophylaxis cohorts only.

    Measured: Up to 79.6 months

  13. Total Quantity of ADAMTS13 Administered During the Treatment of Acute TTP Events in Participants in TAK-755 Treatment Arm

    Total quantity of ADAMTS13 administered during the treatment of acute TTP events (all acute TTP events irrespective of central lab confirmation were included) was assessed. Acute TTP events typically require 3 to 4 days of intensified treatment. As per planned analysis, data for this outcome measure were collected and reported only for the TAK-755 treatment arm of both the prophylaxis (irrespective of the prophylaxis periods) and on demand cohorts.

    Measured: Up to 79.6 months

  14. Incremental Recovery (IR) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    ADAMTS13 activity was measured by the fluorescent resonance energy transfer (FRETS) assay. IR was defined as body weight normalized maximum increase in plasma ADAMTS13 activity level. IR of ADAMTS13 activity for SoC agent and TAK-755 in plasma was assessed. (IU/mL)/(IU/kg) stands for (International units per milliliter)/(International units per kilogram). PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1, end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  15. IR of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    ADAMTS13 antigen was measured using a commercial ADAMTS13 enzyme-linked immunosorbent assay (ELISA) employing ADAMTS13 antigen. IR was defined as body weight normalized maximum increase in plasma ADAMTS13 antigen. IR of ADAMTS13 antigen for SoC agent and TAK-755 in plasma was assessed. (µg/mL)/ (µg/kg) stands for (microgram per milliliter)/(microgram per kilogram). PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1, end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  16. Area Under the Plasma Curve [AUC]All of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    h*IU/mL denotes for hours*international units per milliliters. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  17. AUCall of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    h*µg/mL denotes for hours*microgram per milliliters. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  18. Terminal Half-Life (t1/2) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  19. Mean Residence Time Extrapolated to Infinity (MRT0-inf) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  20. Clearance (CL) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  21. Volume at Steady State (Vss) of ADAMTS13 Activity and ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  22. Maximum Concentration (Cmax) of ADAMTS13 Activity for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    IU/mL stands for International units per milliliter. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth,Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  23. Cmax of ADAMTS13 Antigen for SoC Agent and TAK-755 in Plasma During Prophylactic Treatment

    µg/mL stands for microgram per milliliter. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1 up to 12), PK-II (Month 12:Day 1 up to 12), and PK-III (Month 19:Day 1 up to 12): Pre-infusion and at multiple timepoints post-infusion up to 288 hours

  24. Change From Baseline in Assessment of Von Willebrand Factor:Antigen (VWF:Ag) During Prophylactic Treatment

    VWF:Ag is a measure of total VWF protein and was assessed using a sandwich ELISA employing polyclonal anti-human-VWF antibodies. Assessments of VWF:Ag at baseline and following infusion of the SoC agent and TAK-755 treatment during the initial PK assessment were reported. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 12), PK-II (Month 12:Day 12), and PK-III (Month 19:Day 12): Post-infusion at 288 hours

  25. Change From Baseline in Assessment of Von Willebrand Factor:Ristocetin Cofactor Activity (VWF:RCo) During Prophylactic Treatment

    VWF:RCo provides a measure of the ability of VWF to bind platelet glycoprotein Ib. Stabilized platelets are agglutinated in the presence of VWF and the antibiotic Ristocetin. Assessments of VWF:RCo at baseline and following infusion of the SoC agent and TAK-755 treatment during the initial PK assessment was reported. PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 12), PK-II (Month 12:Day 12), and PK-III (Month 19:Day 12): Post-infusion at 288 hours

  26. Assessment of ADAMTS13 Activity Expressed as Pre-Infusion ADAMTS13 Levels

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  27. Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:RCo

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  28. Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:Ag

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  29. Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Resolution (Res.) Intermediate

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  30. Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Large

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  31. Assessment of Select VWF Parameters Expressed as Pre-Infusion Levels of VWF:mm Low Res. Small

    PK-I, PK-II, and PK-III denote the crossover PK evaluation of a maximum of 14 days at the start of Prophylaxis Treatment Period 1 and end of Prophylaxis Treatment Periods 2 and 3 respectively. As per planned analysis, data for this outcome measure were collected and reported as per the treatment (intervention) received (rADAMTS13 manufactured in Orth, Austria [TAK-755 ORT], rADAMTS13 manufactured in Singapore [TAK-755 SIN], or SoC) during the course of the study, only for the prophylaxis cohorts. No participants received SoC in PK-II and PK-III thus there is no data for the same.

    Measured: PK-I (Month 1:Day 1), PK-II (Month 12:Day 1), and PK-III (Month 19:Day 1): Pre-infusion (within 1 hour)

  32. Number of Participants With Total Binding Antibodies to ADAMTS13 During Prophylactic Treatment

    Total binding antibodies to ADAMTS13 were measured by an ELISA-based assay, detecting total immunoglobulins (IgG, IgA, and IgM). As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.

    Measured: Up to 79.6 months

  33. Number of Participants With Neutralizing Antibodies to ADAMTS13 During Prophylactic Treatment

    Neutralizing antibodies were measured by a Bethesda method with Nijmegen modification using the ADAMTS13 FRETS-VWF73 activity assay. As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.

    Measured: Up to 79.6 months

  34. Number of Participants With Anti-Chinese Hamster Ovary (Anti-CHO) Protein Antibodies During Prophylactic Treatment

    Total immunoglobulin antibodies (Immunoglobulin G [IgG], A [IgA], and M [IgM]) against CHO protein were analyzed using ELISA assay. As per planned analysis, data for this outcome measure were collected and reported per sequence (Prophylaxis Cohort I: TAK-755 Then SoC and Prophylaxis Cohort II: SoC Then TAK-755) for the prophylaxis cohorts only.

    Measured: Up to 79.6 months

  35. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in cTTP-Patient Experience Questionnaire (cTTP-PEQ) Total Score

    The cTTP-PEQ consists of 26 questions designed to assess the participant's experience of fatigue, joint, muscle, abdominal \&chest pain in the previous 24 hours, neurologic manifestations, bruising, feelings of depression and mood alterations, and activity limitation in the past 7 days, and participant's attitudes, experienced side effects, work/school absences and travel impact associated with treatment received for TTP during the previous 2 weeks. The cTTP PEQ is focused on measuring the symptoms and impacts of disease. The total scores range from 0 to 162. A higher score indicates greater burden and poor quality of life. As per planned analysis,for the prophylaxis cohorts the data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per age groups,≥12 years,12 to 18 years,≥18 years for both on demand(OD) and prophylaxis cohorts. No participants in the OD Cohorts had cTTP-PEQ data available for analysis at scheduled post-baseline visits.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  36. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Physical and Mental Component Scores of the 36-Item Short Form Health Survey Version 2 (SF-36v2)

    SF-36v2 is questionnaire that evaluated participant's health related quality of life. It included 36 questions related to 8 health dimensions: physical functioning, role-physical(role limitations due to physical health problems), bodily pain, general health, vitality(energy/fatigue),social functioning, role-emotional(role limitations due to emotional problems),\& mental health. Based on these 4 scales(physical functioning, role-physical, bodily pain, general health), physical component score was generated which ranges between 0 \&100, with higher scores indicating a better quality of life. Based on these 4 scales(vitality, social functioning, role-emotional,\&mental health), mental component score was generated ranging between 0\&100, with higher scores=better quality of life. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected\&reported by categorizing as per Prophylaxis Periods and per component scores for both on demand\&prophylaxis cohorts.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  37. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Abbreviated 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9) Domain Scores

    TSQM is a treatment satisfaction measure used to assess the overall level of participant's satisfaction or dissatisfaction with their medications. TSQM-9 is a 9-item, validated, self-administered instrument used to assess participant's satisfaction with medication. The three domains assessed are treatment effectiveness, convenience, and global satisfaction. The score of each of the 3 domains is based on an algorithm to create a score of 0 to 100. Higher score indicates greater satisfaction in that domain. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  38. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EuroQoL 5 Dimensions Questionnaire 3-Level (EQ-5D-3L) Domain Scores

    EQ-5D-3L health questionnaire is a participant-answered questionnaire scoring 5 dimensions(domains) - mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Each dimension is scored on an ordinal scale with 3 available levels of response and scores ranging from 1 to 3, "no problems," "some problems," and "extreme problems," respectively. Lower scores for the domains in the EQ-5D-3L indicate improvement. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  39. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in EQ-5D-youth (EQ-5D-Y) Domain Scores

    EQ-5D-Y health questionnaire is a participant answered questionnaire scoring 5 dimensions (domains) - mobility, self-care, usual activities, pain/discomfort and anxiety/depression assessed in participants aged from 8 to 16 years. The EQ-5D-Y descriptive system includes 5 descriptive items: Mobility, self-care, doing usual activities, having pain or discomfort, and feeling anxiety or depressed. Each dimension is scored at 3 levels: 1=No problems, 2=some problems, and 3=a lot of problems. Lower scores for the domains in the EQ-5D-Y indicate improvement. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per domain scores for both on demand and prophylaxis cohorts.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  40. Health Related Quality of Life (HRQoL) Assessed as Change From Baseline in Pediatric Quality of Life Inventory (Peds QL) Scale Total Scores

    The PedsQL is a generic health related quality of life instrument designed specifically for a pediatric population and captures following domains: physical functioning, emotional functioning, social functioning, school functioning, psychosocial summary, physical health and total score. The Peds-QL total score consists of all 23 items of all domains. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. Higher scores indicate better quality of life. As per planned analysis, for the prophylaxis cohorts data for this outcome measure were collected and reported by categorizing as per Prophylaxis Periods and per age groups, 2 to < 5 years, 5 to < 8 years, 8 to < 13 years, and 13 to < 18 years, for both on demand and prophylaxis cohorts.

    Measured: Baseline, Urgent Treatment Period: Day 7, End of Period 1 (Month 6), End of Period 2 (Month 12), and End of Period 3 (Month 19)

  41. Resource Utilization: Annualized Length of Hospital Stay for Acute TTP Events for Prophylaxis Cohorts

    The annualized number of days participants stayed in hospital for acute TTP events were assessed. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.

    Measured: Up to 79.6 months

  42. Resource Utilization: Annualized Number of Acute Care Visits for Prophylaxis Cohorts

    Annualized number of acute care visits was calculated as the number of acute care visits × 365.25/(End date - treatment start date + 1). As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.

    Measured: Up to 79.6 months

  43. Resource Utilization: Annualized Number of Days Missed From School or Work for Prophylaxis Cohorts

    Annualized number of days missed from school or work were assessed. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts in a combined manner for Periods 1 and 2 for SoC treatment and for Periods 1, 2, and 3 for TAK-755 treatment respectively.

    Measured: Up to 79.6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted9 Jan 2018
  2. Study start13 Oct 2017
  3. Primary completion28 Dec 2023
  4. Study completion30 May 2024

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Prevention
Allocation
Randomised
Intervention model
Crossover
Model description
This is a Phase 3, prospective, randomized, controlled, open-label, multicenter, 2-period crossover study with a single arm continuation evaluating the safety and efficacy of BAX 930 in the prophylactic and on-demand treatment of participants with severe congenital thrombotic thrombocytopenic purpura (cTTP).
Masking
None (open label)
Enrolment
52 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03393975
Sponsor's protocol ID
281102
Other IDs
2017-000858-18, TAK-755-281102, 2017
Status last verified
Jan 2026
First submitted
5 Oct 2017
Last updated
9 Feb 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Publications

  • Scully M, Antun A, Cataland SR, Coppo P, Dossier C, Biebuyck N, Hassenpflug WA, Kentouche K, Knobl P, Kremer Hovinga JA, Lopez-Fernandez MF, Matsumoto M, Ortel TL, Windyga J, Bhattacharya I, Cronin M, Li H, Mellgard B, Patel M, Patwari P, Xiao S, Zhang P, Wang LT; cTTP Phase 3 Study Investigators. Recombinant ADAMTS13 in Congenital Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2024 May 2;390(17):1584-1596. doi: 10.1056/NEJMoa2314793.

    PubMed 38692292

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing TAK-755 in people with Thrombotic Thrombocytopenic Purpura (TTP), run by Baxalta now part of Shire at 33 sites in 9 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 13 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 33 sites in United States, Spain, France, Italy, Japan and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Mar 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment (time frame: Up to 74.5 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03393975, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2026.

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