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Immune Thrombocytopenia (ITP)Phase 2NCT01054443

A Study to Investigate the Efficacy and Safety of Lusutrombopag (S-888711) Tablets Administered to Adults With Immune Thrombocytopenia (ITP)

A Phase 2 study testing Lusutrombopag in people with Immune Thrombocytopenia (ITP), run by Shionogi at 19 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

The primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Immune Thrombocytopenia (ITP)
What is being tested?
Lusutrombopag
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
20 participants
Where
19 sitesUnited States
Study dates
18 Mar 2010to 24 Nov 2010
Phase
Phase 2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • A signed and dated written informed consent
  • Males and females ≥ 18 years of age
  • All subjects must agree to use barrier contraception
  • Diagnosis of ITP
  • Subjects > 60 years must have had a diagnostic bone marrow aspiration
  • Relapsed persistent or chronic ITP status, with or without prior splenectomy (exception: in Hungary only splenectomized subjects will be enrolled), after having failed at least 1 prior ITP therapy (excluding TPO agonists) and have a platelet count < 30,000/μL if not taking medications or < 50,000/μL despite concomitant steroids or other ITP therapies, such as danazol or immunosuppressive drugs
Show all 9Show fewer
  • Subjects receiving steroid therapy must be on a stable dose
  • Prothrombin time (PT) and activated partial thromboplastin time (aPTT) within 20% of the upper limit of normal (ULN)
  • Subjects receiving stable dosages of cyclosporine A, mycophenolate mofetil, azathioprine, or danazol are allowed. The dosages of all these medications must be stable for at least 4 weeks prior to Visit 1 (Day 1)

Who cannot join

  • History of clinically important hemorrhagic clotting disorder
  • Females who are pregnant, lactating, or taking oral contraceptives
  • History of alcohol/drug abuse or dependence within 1 year
  • Use of the following drugs or treatment prior to Visit 1 (Day 1):
  • Within 12 weeks - alemtuzumab, multi-drug systemic chemotherapy, stem cell therapy;
  • Within 8 weeks - rituximab
Show all 26Show fewer
  • Within 2 weeks - platelet transfusions or plasmapheresis treatment
  • Within 4 weeks - use of anti-platelet or anti-coagulant drugs
  • Within 1 week - Rho(D) immune globulin or intravenous immunoglobulin
  • History of clinically significant cardiovascular or thromboembolic disease within 26 weeks prior to Screening
  • Splenectomy within 4 weeks prior to Screening
  • Clinically significant laboratory abnormalities
  • Hemoglobin < 10.0 g/dL for men or women, not clearly related to ITP
  • Absolute neutrophil count < 1000/mm^3
  • Abnormal peripheral blood smear
  • Total bilirubin > 1.5 x upper limit of normal
  • Alanine aminotransferase (ALT) > 1.5 x upper limit of normal
  • Aspartate aminotransferase (AST) > 1.5 x upper limit of normal
  • Creatinine > 1.5 x upper limit of normal
  • Human immunodeficiency virus (HIV) positive
  • Hepatitis A immunoglobulin M antibody (IgM HAV) positive, hepatitis B surface antigen (HbsAg) or hepatitis C antibody (HCV) positive
  • Thyroid stimulating hormone (TSH) > 1.5 x upper limit of normal
  • Free thyroxine (T4) > 1.5 x upper limit of normal
  • Exposure to previous thrombopoietin (TPO) mimetics/agonists (e.g., eltrombopag,romiplostim, E5501 [AKR-501] or LGD-4665) within 4 weeks prior to Screening
  • Subjects unresponsive to previous TPO mimetics/agonists (e.g., eltrombopag, romiplostim, E5501 [AKR-501] or LGD-4665)
  • Exposure to an investigative medication within the past 30 days

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

United States19 sites

  • Investigator

    Anaheim, California, 92801

  • Investigator

    Atlanta, Georgia, 30341

  • Investigator

    Bethesda, Maryland, 20817

  • Investigator

    Boston, Massachusetts, 02114

  • Investigator

    Boynton Beach, Florida, 33426

  • Investigator

    Cleveland, Ohio, 44106

  • Investigator

    Jacksonville, Florida, 32207

  • Investigator

    Jefferson City, Missouri, 65109

  • Investigator

    Kansas City, Missouri, 64131

  • Investigator

    Los Angeles, California, 90272

  • Investigator

    Metairie, Louisiana, 70006

  • Investigator

    New Brunswick, New Jersey, 08903

  • Investigator

    New York, New York, 10021

  • Investigator

    New York, New York, 10029

  • Investigator

    Riverdale, Georgia, 30274

  • Investigator

    Salt Lake City, Utah, 84132

  • Investigator

    San Antonio, Texas, 78229

  • Investigator

    Seattle, Washington, 98109

  • Investigator

    Washington D.C., District of Columbia, 20007

Other trials in United States

About this study

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Investigate the Efficacy and Safety of S-888711 Tablets Administered Once-daily for 42 Days to Adult Subjects With Relapsed Persistent or Chronic Immune Thrombocytopenia With or Without Prior Splenectomy

The primary objective of this study was to assess the efficacy of 3 dose levels of lusutrombopag (0.5 mg, 0.75 mg, and 1.0 mg) and placebo on platelet count.

Being tested
Placebo, Lusutrombopag
Keywords
Blood Platelet Disorders, Immune Thrombocytopenia (ITP), Low Platelet Count, Thrombocytopaenia, S-888711, Splenectomy, Thrombopoiesis, Hematologic Disease, Auto-immune thrombocytopenic Purpura, Relapsed Persistent or Chronic ITP, Idiopathic Thrombocytopenic Purpura, Thrombotic Thrombocytopenic Purpura (TTP)
Medical subject headings
Purpura, Thrombocytopenic, Idiopathic, Blood Platelet Disorders, Thrombocytopenia, Hematologic Diseases, Purpura, Thrombotic Thrombocytopenic

What participants receive

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

  • PlaceboDrug

    Tablet

  • LusutrombopagDrug

    Also called MULPLETA®, S-888711

    Tablet

Study groups in NCT01054443
Study groupRoleWhat they receive
PlaceboPlacebo comparatorParticipants received placebo tablets orally once a day for 42 days.
Receives: Drug: Placebo
Lusutrombopag 0.5 mgExperimentalParticipants received 0.5 mg lusutrombopag orally once a day for 42 days.
Receives: Drug: Lusutrombopag
Lusutrombopag 0.75 mgExperimentalParticipants received 0.75 mg lusutrombopag orally once a day for 42 days.
Receives: Drug: Lusutrombopag
Lusutrombopag 1.0 mgExperimentalParticipants received 1.0 mg lusutrombopag orally once a day for 42 days.
Receives: Drug: Lusutrombopag

What is being measured

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

  1. Percentage of Participants With a Response

    Responders were participants with one of the following:

    1. achieved a platelet count of ≥ 50,000 cells/µL after 6 weeks of dosing; or
    2. prematurely withdrawn due to a platelet count > 400,000 cells/µL prior to Day 42.

    Participants were counted as non-responders if any of the following conditions held:

    • The above conditions were not satisfied;
    • They received rescue medications;
    • They satisfied the above conditions after receiving restricted medications during the treatment period;
    • They had achieved a platelet count of ≥ 50,000 cells/µL before Week 6 but not after Week 6; or
    • They withdrew for any reason other than a platelet count > 400,000 cells/µL.

    Measured: Week 6

Secondary outcomes (9)

  1. Change From Baseline in Platelet Count at Week 6

    Measured: Baseline and Week 6

  2. Duration of Response

    Duration of response was defined as the percentage of the cumulative time a platelet count was ≥ 50,000 cells/µL during the treatment period.

    Measured: 6 weeks

  3. Percentage of Participants Who Achieved a Platelet Count of ≥ 30,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing

    Measured: Week 6

  4. Percentage of Participants Who Achieved a Platelet Count of ≥ 50,000 Cells/µL and Doubled the Baseline Platelet Count After 6 Weeks of Dosing

    Measured: Week 6

  5. Number of Participants With Worst Severity of Bleeding Associated With ITP During the Treatment Period,

    Bleeding assessments were performed by the Investigator according to the World Health Organization (WHO) criteria bleeding scale:

    Grade 0: no bleeding; Grade 1: petechial bleeding; Grade 2: mild blood loss (clinically significant); Grade 3: gross blood loss, requires transfusion (severe); Grade 4: debilitating blood loss, retinal or cerebral associated with fatality.

    For each participant, the most severe WHO bleeding grade observed during the 6-week treatment period is reported.

    Measured: 6 weeks

  6. Number of Participants Who Received Rescue Medication During the Treatment Period

    Measured: 6 weeks

  7. Number of Participants With Adverse Events (AEs)

    An AE is defined as any untoward medical occurrence in a subject administered a pharmaceutical product during the course of a clinical investigation, including any unfavorable and unintended sign, symptom, or disease temporally associated with the use of an investigational product (IP), whether or not thought to be related to the IP. AEs reported after initial study drug administration were considered treatment-emergent.

    A serious adverse event is defined as any AE that resulted in death, was life-threatening, hospitalization or prolongation of existing hospitalization, a persistent or significant disability/incapacity, or a congenital anomaly/birth defect or an important medical event that, based upon medical judgment, may jeopardize the participant or require medical or surgical intervention to prevent one of the outcomes listed above.

    A treatment-related AE is any AE determined by the investigator to be possibly related, probably related, or definitely related to study drug.

    Measured: 6 weeks

  8. Lusutrombopag Plasma Concentration

    Plasma concentrations of lusutrombopag were determined using a validated liquid chromatography mass spectrometry method. The lower limit of quantification (LOQ) for the plasma assay for lusutrombopag was 0.1 ng/mL.

    Measured: Days 8, 22, and 36, after dosing

  9. Plasma Concentration of Metabolite S-888711 Deshexyl

    Plasma concentrations of the major metabolite S-888711 deshexyl were determined using a validated liquid chromatography mass spectrometry method. The lower limit of quantification (LOQ) for the plasma assay for S-888711 deshexyl was 0.1 ng/mL.

    Measured: Days 8, 22, and 36, after dosing

Timeline

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

  1. First posted22 Jan 2010 (estimated)
  2. Study start18 Mar 2010
  3. Primary completion24 Nov 2010
  4. Study completion24 Nov 2010

Why it stopped: This study was terminated before the planned sample size was enrolled because results indicated that a higher dose was necessary to elicit an efficacy effect.

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 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
20 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01054443
Sponsor's protocol ID
0913M0621
Status last verified
Feb 2021
First submitted
20 Jan 2010
Last updated
18 Mar 2021
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Lusutrombopag in people with Immune Thrombocytopenia (ITP), run by Shionogi at 19 sites in United States.

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 9 inclusion and 26 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: stopped early, as of the sponsor's last update on 18 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 19 sites in United States. 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 24 Feb 2021). 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?

Percentage of Participants With a Response (time frame: Week 6). 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 NCT01054443.

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 18 Mar 2021.

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