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    Summary
    EudraCT Number:2022-003501-29
    Sponsor's Protocol Code Number:161503
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-12-13
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2022-003501-29
    A.3Full title of the trial
    Efficacy, Safety, Tolerability, Immunogenicity and Pharmacokinetic Evaluation of HYQVIA in Pediatric Subjects with Primary Immunodeficiency Diseases
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of HYQVIA in Children with Primary Immunodeficiency Diseases
    A.4.1Sponsor's protocol code number161503
    A.5.4Other Identifiers
    Name:ClinicalTrials.govNumber:NCT03277313
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBaxalta Innovations GmbH
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBaxalta Innovations GmbH
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta Innovations GmbH
    B.5.2Functional name of contact pointClinical Trial Transparency
    B.5.3 Address:
    B.5.3.1Street AddressIndustriestrasse 67
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1221
    B.5.3.4CountryAustria
    B.5.6E-mailClinicalTransparency@takeda.com
    B.Sponsor: 2
    B.1.1Name of SponsorBaxalta US Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportBaxlta US Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBaxalta US Inc.
    B.5.2Functional name of contact pointClinical Trial Transparency
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.6E-mailClinicalTransparency@takeda.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name HyQvia 100 mg/ml solution for infusion for subcutaneous use
    D.2.1.1.2Name of the Marketing Authorisation holderBaxalta Innovations GmbH
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameHyQvia
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Yes
    D.3.11.7Plasma derived medicinal product Yes
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Immunodeficiency Diseases (PID)
    E.1.1.1Medical condition in easily understood language
    Conditions due to defects of the immune system
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of HYQVIA treatment in pediatric subjects with PIDD who received prior IV or SC immunoglobulin therapy before enrollment into the study.
    E.2.2Secondary objectives of the trial
    Secondary objectives of the study are further efficacy and safety assessments (e.g., immunogenicity), tolerability, characteristics of product administration, treatment preference and satisfaction, health-related quality of life, and PK parameters.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject 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.
    2. Subject is at least two and below 16 years of age at the time of screening.
    3. Subject has been receiving a consistent dose of 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 BW / 4 weeks.
    4. Subject has a serum trough level of IgG > 5 g/L at screening.
    5. If female of childbearing potential, subject presents with a negative pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
    6. Subject/legally authorized representative is willing and able to comply with the requirements of the protocol.
    E.4Principal exclusion criteria
    1. Subject 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.
    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):
    a. Persistent alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 2.5 times the upper limit of normal (ULN) for the testing laboratory
    b. Persistent severe neutropenia (defined as an absolute neutrophil count [ANC] ≤ 500/mm3)
    3. Subject has anemia that would preclude phlebotomy for laboratory studies, according to standard practice at the site.
    4. Subject has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IV immunoglobulin, SC immunoglobulin, and/or Immune Serum Globulin (ISG) infusions.
    5. Subject has severe IgA deficiency (less than 7.0 mg/dL) with known anti-IgA antibodies and a history of hypersensitivity.
    6. Subject has a known allergy to hyaluronidase.
    7. Subject has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
    8. Subject 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.
    9. Subject has severe dermatitis that would preclude adequate sites for safe product administration in the opinion of the investigator.
    10. Subject 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.
    11. Subject is a family member or employee of the investigator.
    12. If female, subject is pregnant or lactating at the time of enrollment.
    E.5 End points
    E.5.1Primary end point(s)
    Rate of acute serious bacterial infections
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study period of approximately 6 years
    E.5.2Secondary end point(s)
    Efficacy
    1. Number of all infections per patient-year
    2. Trough levels of IgG and IgG subclasses for Study Epoch 2
    3. Trough levels of specific antibodies to clinically relevant pathogens (Clostridium tetani toxoid, Haemophilus influenzae, and Hepatitis B Virus [HBV]) for Study Epoch 2

    Pharmacokinetics
    1. For the PK assessment in Epoch 2 the following PK parameters will be determined: area under the curve (AUC), clearance (CL), maximum concentration (Cmax), minimum concentration (Cmin), time to maximum concentration (Tmax), and terminal half-life

    Safety
    1. Number and rate per infusion (excluding infections) of SAEs, related and not related
    2. Number and rate per infusion (excluding infections) of all AEs, related and not related
    3. Number and rate per infusion (excluding infections) of local AEs, related and not related
    4. Number and rate per infusion (excluding infections) of systemic AEs, related and not related
    5. Number and rate per infusion (excluding infections) of all temporally associated AEs (begin during or within 72 hours of completion of infusion)
    6. Number and rate per infusion (excluding infections) of all causally related and/or temporally associated AEs
    7. Rates of all AEs (excluding infections) defined as number of AEs categorized by MedDRA preferred terms, seriousness, and severity, divided by the number of infusions
    8. Number/proportion of subjects who develop positive titer (≥ 160) of binding or neutralizing antibodies to rHuPH20

    Mode of Product Administration
    1. Infusions (Study Epoch 2)
    a. Number of infusions per month
    b. Number of infusion sites (needle sticks) per infusion/month
    c. Duration of infusion
    d. Maximum infusion rate / site
    e. Infusion volume / site
    f. Number/proportion of infusions that are discontinued, slowed, or interrupted due to an AE
    2. Number of weeks to reach final dose interval (three weeks or four weeks) (Epoch 1)
    3. Proportion of subjects who achieve a treatment interval of three or four weeks in Epoch 2
    4. Proportion of subjects who maintain a treatment interval of three or four weeks in Epoch 2 for 12 months

    Health-related Quality of Life (HRQoL)
    Assessment of Health-related Quality of Life (HRQoL) Questionnaire:
    1. Pediatric Quality of Life Inventory (Peds-QL)
    2. EuroQol five dimensions questionnaire (EQ-5D)

    Treatment Preference and Satisfaction
    1. Assessment of Life Quality Index (LQI)
    2. Assessment of Treatment Satisfaction and Medication Questionnaire (TSQM-9)
    3. Assessment of Treatment Preference Questionnaire

    Health Resource Utilization
    1. Days not able to go to school or work, or to perform normal daily activities due to infection or other illnesses per patient-year
    2. Days on antibiotics per patient-year
    3. Number of hospitalizations, indication for the hospitalization (infection or non-infection) and days hospitalized per patient-year
    4. Number of acute physician visits (office and emergency room) due to infection or other illnesses per patient-year
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy:
    1: Throughout the study period of approx. six years
    2: Epoch 2, Months 0, 6, 12, 18, 24, 30, 36
    3: Epoch 2
    PK:
    Pre-infusion, Post-infusion (Day 2 (≥12 years of age), Days 4, 10, day 21 (end of PK for 3 week interval), day 28 (end of PK for 4 week interval)
    Safety:
    1-7: Throughout the study period of approx. six years
    8: Epoch 1 (up to 6 weeks) and Epoch 2 (up to 3 years)
    Mode of Product Administration:
    1: Epoch 2 (up to 3 years)
    2: Epoch 1 (up to 6 weeks)
    3: 3 or 4 weeks (dependent on treatment interval)
    4: 12 months
    HRQol:
    1-2: Epoch 1: Baseline (First Infusion); Epoch 2: months 12, 24, 36
    Treatment:
    1-2: Epoch 1: Baseline (First Infusion); Epoch 2: months 12, 24, 36
    3: Epoch 2: months 12, 24, 36
    HRU:
    1-4: Throughout the study (approx. six years)
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 44
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 32
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    parent/legal representative to provide consent for pediatric patients
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 40
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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