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Primary Immunodeficiency Diseases (PID)Phase 3NCT03277313

Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric PIDD Subjects

A Phase 3 study testing HYQVIA in people with Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire at 19 sites in United States.

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

The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary immunodeficiency disease (PIDD). Read more about this study

Who is it for?
Ages 2 to 15 years
Conditions the study is for
Primary Immunodeficiency Diseases (PID)
What is being tested?
HYQVIA
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
44 participants
Where
19 sitesUnited States
Study dates
25 Sep 2017to 20 Jul 2022
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 2 to 15 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Participant must have a documented diagnosis of a form of primary immunodeficiency (PI) involving a defect in antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies (IUIS) Scientific Committee 2015 (Picard et al., 2015) prior to enrollment. The diagnosis must be confirmed by the sponsor´s Medical Director prior to first treatment with IP in the study.
  • Participant is at least two and below 16 years of age at the time of screening.
  • Participant has been receiving a consistent dose of Immunoglobulin G (IgG), administered in compliance with the respective product information for a period of at least three months prior to screening. The average minimum pre-study dose over that interval was equivalent to 300 mg/kg BW / 4 weeks and a maximum dose equivalent to 1000 mg/kg body weight (BW) / 4 weeks.
  • Participant has a serum trough level of IgG > 5 g/L at screening.
  • If female of childbearing potential, participant presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
  • Participant /legally authorized representative is willing and able to comply with the requirements of the protocol.

Who cannot join

  • Participant has a known history of or is positive at screening for one or more of the following: hepatitis B surface antigen (HBsAg), polymerase chain reaction (PCR) for hepatitis C virus (HCV), PCR for human immunodeficiency virus (HIV) Type 1/2.
  • Abnormal laboratory values at screening meeting any one of the following criteria (abnormal tests may be repeated once to determine if they are persistent):
  • Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 2.5 times the upper limit of normal (ULN) for the testing laboratory
  • Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] ≤ 500/mm^3)
  • Participant has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site.
  • Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following intravenous (IV) immunoglobulin, subcutaneous (SC) immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
Show all 14Show fewer
  • Participant has severe Immunoglobulin A (IgA) deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity.
  • Participant has a known allergy to hyaluronidase.
  • Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
  • Participant has a bleeding disorder or a platelet count less than 20,000/μL, or who, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of SC therapy.
  • Participant has severe dermatitis that would preclude adequate sites for safe product administration in the opinion of the investigator.
  • Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • Participant is a family member or employee of the investigator.
  • If female, participant is pregnant or lactating at the time of enrollment.

Trial locations and contacts

19 sites in 1 country.

19 sites in 1 country

United States19 sites

  • Georgia Pollens Clinical Research Centers, Inc.

    Albany, Georgia, 31707

  • Emory Healthcare

    Atlanta, Georgia, 30322

  • University of Alabama Medical Center

    Birmingham, Alabama, 35294

  • Boston Children's Hospital

    Boston, Massachusetts, 02115

  • Women & Children's Hospital of Buffalo

    Buffalo, New York, 14203

  • Marshall University Joan C. Edwards School of Medicine

    Charleston, West Virginia, 25701

  • Carolinas Healthcare System

    Charlotte, North Carolina, 28203

  • Allergy Asthma & Immunology Relief of Charlotte

    Charlotte, North Carolina, 28277

  • Allergy Partners of North Texas Research

    Dallas, Texas, 75230

  • Section on Immunopathogenesis and Clinical Immunology

    Fairfax, Virginia, 22030

  • Northwell Health, Inc. PRIME

    Great Neck, New York, 11021

  • Connecticut Children's Medical Center

    Hartford, Connecticut, 06106

  • University of Miami Pediatric Allergy and Immunology

    Miami, Florida, 33136

  • OK Institute of Allergy & Asthma Clinical Research, LLC

    Oklahoma City, Oklahoma, 73131

  • University of Utah

    Salt Lake City, Utah, 84132

  • Washington University

    St Louis, Missouri, 63110

  • University of South Florida Physician Group

    St. Petersburg, Florida, 33701

  • Stony Brook Children's Hospital

    Stony Brook, New York, 11794

  • Vital Prospects Clinical Research Institute, P.C.

    Tulsa, Oklahoma, 74136

Other trials in United States

About this study

Official title: Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects With Primary Immunodeficiency Diseases

The purpose of the study is to acquire additional data on efficacy, safety, tolerability, immunogenicity, pharmacokinetic (PK) and other parameters of HYQVIA in pediatric (age ≥ 2 to <16 years) participants with primary immunodeficiency disease (PIDD).

Being tested
HYQVIA
Medical subject headings
Primary Immunodeficiency Diseases

What participants receive

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

  • HYQVIABiological

    Also called IGI 10% with rHuPH20

    Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase (IGI, 10% with rHuPH20)

Study groups in NCT03277313
Study groupRoleWhat they receive
Epoch 1ExperimentalPediatric participants with PIDD who were on IV or non-HYQVIA SC treatment with immunoglobulin were enrolled and treated with HYQVIA SC with a dose or interval ramp-up period of up to six weeks. HYQVIA dose was planned to be equivalent to 100% (± 5%) of pre-study treatment. Dose frequency was one treatment interval of one week, then one treatment interval of two weeks for participants who were planned to be treated every three weeks, and one more treatment interval of three weeks for participants who were planned to be treated every four weeks.
Receives: Biological: HYQVIA
Epoch 2ExperimentalEpoch 1 was followed by Epoch 2 with HYQVIA treatment infusions given once every 3 or 4 weeks, depending on the participant's previous IV dosing schedule (for IV pretreated participants) and at the discretion of the investigator and participant (for SC-pretreated participants) up to approximately 36 months.
Receives: Biological: HYQVIA

What is being measured

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

  1. Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year

    The rate of ASBI was defined as the mean number of ASBI per participant-year based on the United States (U.S.) Food and drugs Administration (FDA) guidance for industry to support marketing of human immune globulin intravenous (IGIV) as replacement therapy for primary humoral immunodeficiency and the European Medicines Agency (EMA) guideline on the clinical investigation of human normal immunoglobulin for SC and /or intramuscular administration. ASBI included bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, diagnosed according to the Diagnostic Criteria for Acute Serious Bacterial Infections.

    Measured: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

Secondary outcomes (46)

  1. Rate Represented as Mean Number of All Infections Per Participant-year

    The rate of all infections was defined as the mean number of all infections per participant-year. Number of all infections was calculated as number of infections per participant-year.

    Measured: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 months)

  2. Epoch 2: Trough Levels of Immunoglobulin G (IgG) Total and IgG Subclasses

    Measured: Study Epoch 2, Year 1: Months 0, 6, and 12; Year 2: Months 18, 24 and 36

  3. Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens Categorized as Clostridium Tetani Toxoid and Hepatitis B Virus

    Measured: Study Epoch 2, Year 2: Months 6, 24, and 36

  4. Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogen Haemophilus Influenzae B

    Measured: Study Epoch 2, Year 2: Months 6, 24, and 36

  5. Epoch 2: Area Under the Curve Normalized for Week (AUCweek)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  6. Epoch 2: Area Under the Curve Over the Infusion Interval (AUCTau)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  7. Epoch 2: Apparent Clearance (CL/F)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  8. Epoch 2: Maximum Concentration (Cmax)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  9. Epoch 2: Minimum Concentration (Cmin)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  10. Epoch 2: Time to Maximum Concentration (Tmax)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  11. Epoch 2: Terminal Half-life (T 1/2)

    Measured: Study Epoch 2, Month 6: Day 0 pre-infusion, and at Days 2, 4, 10, 21 and 28 post-infusion

  12. Number of Participants With Serious Adverse Events (SAEs) Excluding Infections, Related and Not Related

    An SAE is defined as an untoward medical occurrence that at any dose is fatal, life-threatening, requires inpatient hospitalization or results in prolongation of an existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, is a medically important event. Number of participants who experienced SAEs, related or not related, was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  13. Rate of SAEs Excluding Infections, Related and Not Related, Per Infusion

    Rate of SAEs per infusion was calculated as number of serious adverse events/total number of infusions administered to participants in the analysis set. Rate of SAEs per infusion (excluding infections) was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  14. Number of Participants With All Treatment Emergent Adverse Events (TEAEs) Excluding Infections, Related and Not Related

    An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced TEAEs, related or not related, was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  15. Rate of All TEAEs Excluding Infections, Related and Not Related, Per Infusion

    Rate of all TEAEs per infusion was calculated as number of any adverse reaction events/total number of infusions administered to participants in the analysis set. Rate of any adverse reactions per infusion (excluding infections) was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  16. Number of Participants With Local TEAEs Excluding Infections, Related and Not Related

    An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced local TEAEs, related or not related, was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  17. Rate of Local TEAEs Excluding Infections, Related and Not Related, Per Infusion

    Rate of local TEAEs per infusion was calculated as number of local TEAEs/total number of infusions administered to participants in the analysis set. Rate of local TEAEs per infusion (excluding infections) was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  18. Number of Participants With Systemic TEAEs Excluding Infections, Related and Not Related

    An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced systemic TEAEs, related or not related, was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  19. Rate of Systemic TEAEs Excluding Infections, Related and Not Related, Per Infusion

    Rate of systemic TEAEs per infusion was calculated as number of systemic TEAEs/total number of infusions administered to participants in the analysis set. Rate of systemic TEAEs per infusion (excluding infections) was reported.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  20. Number of Participants With All Temporally Associated TEAEs Excluding Infections

    An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants who experienced all temporally associated TEAEs, related or not related, was reported.

    Measured: From beginning of infusion up to 72 hours post infusion

  21. Rate of All Temporally Associated TEAEs Excluding Infections Per Infusion

    Rate of all temporally associated TEAEs per infusion was calculated as number of all temporally associated TEAEs/total number of infusions administered to participants in the analysis set. Rate of all temporally associated TEAEs per infusion (excluding infections) was reported.

    Measured: From beginning of infusion up to 72 hours post infusion

  22. Number of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections

    An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Number of participants with any related (causally) and/or temporally associated TEAEs (excluding infections) was reported. Temporally-associated TEAEs were defined as TEAEs which begin during infusion of IP or within 72 hours following the end of IP infusion.

    Measured: From beginning of infusion up to 72 hours post infusion

  23. Rate of Participants With All Related (Causally) and/or Temporally Associated TEAEs Excluding Infections Per Infusion

    Rate of any related (causally) and/or temporally associated TEAEs per infusion was calculated as number of related and/or temporally associated adverse events/ total number of infusions administered to participants in the analysis set. TEAEs recorded in the study database as "possibly related" or "probably related" to HYQVIA are considered HYQVIA-related adverse events. Temporally-associated TEAEs were defined as TEAEs which begin during infusion of IP or within 72 hours following the end of IP infusion. Rate of any related (causally) and/or temporally associated TEAEs per infusion (excluding infections) was reported.

    Measured: From beginning of infusion up to 72 hours post infusion

  24. Percentage of Participants With Any TEAEs Excluding Infections

    An AE is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. A TEAE is defined as an adverse event with an onset that occurs after receiving study drug. Percentages are rounded off to whole number at the nearest decimal.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  25. Number of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20

    Number of participants who developed positive titer (rHuPH20 titer ≥160) of binding antibodies to rHuPH20 was reported. Neutralizing antibodies were only tested if the participant had a titer of ≥160 of binding antibodies. Participants with multiple assessments of titer of ≥160 of binding antibodies are counted only once.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  26. Percentage of Participants Who Developed Positive Titer (≥160) of Binding or Neutralizing Antibodies to rHuPH20

    Percentage of participants who developed positive titer (rHuPH20 titer ≥160) of binding antibodies to rHuPH20 was reported. Neutralizing antibodies were only tested if the participant had a titer of ≥160 of binding antibodies. Participants with multiple assessments of titer of ≥160 of binding antibodies are counted only once. Percentages are rounded off to whole number at the nearest decimal.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  27. Epoch 2: Number of Infusions Per Month

    Measured: Study Epoch 2: Up to 36 months

  28. Epoch 2: Number of Infusion Sites (Needle Sticks) Per Infusion

    Measured: Study Epoch 2: Up to 36 months

  29. Epoch 2: Number of Infusion Sites (Needle Sticks) Per Month

    Measured: Study Epoch 2: Up to 36 months

  30. Epoch 2: Duration of Infusion

    Duration of infusion is calculated as (stop time of infusion - start time of infusion) (in Epoch 2). Duration of infusion is the time from the start of rHuPH20 infusion until the stop time of immunoglobulin infusion.

    Measured: Study Epoch 2: Up to 36 months

  31. Epoch 2: Maximum Infusion Rate Per Site

    HYQVIA treatment infusions in Epoch 2 were given at a rate of 10 milliliters per hour per site (mL/h/site) to 160 ml/h/site (body weight [BW] of <40 kg) and 10 mL/h/site to 300 mL/h/site (BW of ≥40 kg).

    Measured: Study Epoch 2: Up to 36 months

  32. Epoch 2: Infusion Volume Per Site

    Infusion volume per site is calculated as (actual IgG volume (mL) / total number of infusion sites used).

    Measured: Study Epoch 2: Up to 36 months

  33. Epoch 2: Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE

    Measured: Study Epoch 2: Up to 36 months

  34. Epoch 2: Percentage of Infusions Which Were Discontinued, Slowed, or Interrupted Due to an AE

    Percentages are rounded off to whole number at the nearest decimal.

    Measured: Study Epoch 2: Up to 36 months

  35. Epoch 1: Number of Weeks to Reach Final Dose Interval

    Measured: Epoch 1 (up to 6 weeks)

  36. Epoch 2: Percentage of Participants Who Achieved a Treatment Interval of Three or Four Weeks in Epoch 2

    Percentages are rounded off to whole number at the nearest decimal. Percentages may sum up to more than 100% as assigned treatment interval could be changed during the study.

    Measured: Study Epoch 2: Up to 36 months

  37. Epoch 2: Percentage of Participants Who Maintained a Treatment Interval of Three or Four Weeks in Epoch 2 for 12 Months

    Percentages are rounded off to whole number at the nearest decimal.

    Measured: Study Epoch 2: Up to 12 months

  38. Health-related Quality of Life (HRQoL): Change From Baseline in Pediatric Quality of Life Inventory (Peds-QL) Score

    Peds-QL=generic questionnaire developed and validated to measure HRQoL among pediatric population. It included assessment of 4 dimensions: physical functioning (8 items), emotional functioning (8 items), social functioning (8 items), and school functioning (Age groups: Toddler (2-4years),Young child (5-7years),Child (8-12years), and Teens (13-<16years). Depending on the participants age, questionnaire may be completed by participant or parent/caregiver as appropriate. Items were scored on a 5-point Likert scale: 0=never a problem; 1=almost never a problem; 2=sometimes a problem; 3=often a problem; and 4=almost always a problem). All Scores were transformed on a total scale from 0-100 where, 0=100, 1=75, 2=50, 3=25, and 4=0. Higher scores indicating better health status. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

    Measured: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

  39. HRQoL: Change From Baseline in EuroQol Five Dimensions Questionnaire (EQ-5D) Score

    The EQ-5D is a validated, self-administered assessment of overall health consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). Participants were asked to describe their health state that day by choosing 1 of 3 responses that reflect the levels of severity for each of the five dimensions: no problems, some or moderate problems, or extreme problems. The domain scores are calculated with higher scores indicating worsening health status. The EQ-5D also includes a standard vertical 20-cm visual analogue scale (similar to a thermometer) for recording a participant's rating of their current HRQoL state, which ranged from 0 to 100, where 0 indicated worst imaginable health state and 100 was best imaginable health state. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

    Measured: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

  40. Treatment Preference and Satisfaction: Change From Baseline in Assessment of Life Quality Index (LQI) Score

    The LQI is a validated questionnaire assessing participant perceptions of their HRQoL and their treatment specifically among participants who use IgG therapy. This questionnaire covers 4 domains: treatment interferences, therapy-related problems, therapy setting, and treatment costs. The LQI domains are scored from 0 to 100, with higher scores associated with better IgG treatment satisfaction. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

    Measured: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

  41. Treatment Preference and Satisfaction: Change From Baseline in Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9) Score

    The TSQM-9 is a 9-item, validated, self-administered instrument to assess participant satisfaction with medication, which assesses 3 domains: effectiveness, convenience, and global satisfaction. The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction. End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

    Measured: Epoch 1: Baseline (First Infusion); Study Epoch 2: Up to Month 36

  42. Treatment Preference and Satisfaction: Number of Participants Who Completed Treatment Preference Questionnaire

    The treatment preference questionnaire, internally developed by the study sponsor, is a self-administered, non-validated scale assessing participant preference for various attributes of immunoglobulin G (IgG) therapy.End of Epoch 2 summarizes all participant's data for their last epoch 2 visit (so not including the participant that discontinued in epoch 1).

    Measured: Study Epoch 2: Up to Month 36

  43. Health Resource Utilization: Days Not Able to go to School or Work, or to Perform Normal Daily Activities

    Days not able to go to school or work, or to perform normal daily activities due to infection or other illnesses were calculated as days not able to go to school or work, or to perform normal daily activities due to infection or other illnesses per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  44. Health Resource Utilization: Days on Antibiotics

    Days on antibiotics were calculated as days on antibiotics per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  45. Health Resource Utilization: Number of Hospitalizations

    Number of hospitalizations, indication for the hospitalization (infection or non-infection) were calculated as number of hospitalizations, indication for the hospitalization (infection or non-infection) per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

  46. Health Resource Utilization: Number of Days Hospitalized Per Participant-Year

    Number of days hospitalized were calculated as number of days hospitalized per participant-year. Per participant-years = number or days reported / total number of years of study duration, i.e., the sum of study duration for all participants in the analysis set, divided by 365.25.

    Measured: From first dose of study drug up to EOS (up to 4 years 9 months)

Timeline

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

  1. First posted11 Sep 2017
  2. Study start25 Sep 2017
  3. Primary completion20 Jul 2022
  4. Study completion20 Jul 2022

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
44 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03277313
Sponsor's protocol ID
161503
Status last verified
Sep 2023
First submitted
7 Sep 2017
Last updated
23 Oct 2023
Data monitoring committee
No
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

  • Li Z, Teng Y, Russo-Schwarzbaum S, McCoy B. Pharmacokinetics of hyaluronidase-facilitated subcutaneous immunoglobulin 10% in pediatric patients with primary immunodeficiency disease. Immunother Adv. 2026 Apr 3;6(1):ltag003. doi: 10.1093/immadv/ltag003. eCollection 2026.

    PubMed 41937917

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing HYQVIA in people with Primary Immunodeficiency Diseases (PID), run by Baxalta now part of Shire 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 6 inclusion and 14 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 23 Oct 2023. 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. 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 23 Oct 2023). 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?

Rate Represented as Mean Number of Acute Serious Bacterial Infections (ASBI) Per Participant-year (time frame: From first dose of study drug up to end of Study Epoch 2 (up to approximately 37.2 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 NCT03277313, 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 23 Oct 2023.

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