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    Summary
    EudraCT Number:2022-001873-29
    Sponsor's Protocol Code Number:TAK-664-3001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-06-10
    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 number2022-001873-29
    A.3Full title of the trial
    A Phase 3, Open-label, Non-controlled, Multi-dose Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Subjects with Primary Immunodeficiency Diseases (PID)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
    A.4.1Sponsor's protocol code numberTAK-664-3001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04346108
    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 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
    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
    B.Sponsor: 2
    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
    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 code1220
    B.5.3.4CountryAustria
    B.5.6E-mailClinicalTransparency@takeda.com
    B.Sponsor: 3
    B.1.1Name of SponsorTakeda Pharmaceutical Company Limited
    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 supportTakeda Pharmaceutical Company Limited
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda
    B.5.2Functional name of contact pointClinical Trial Transparency
    B.5.3 Address:
    B.5.3.1Street Address95 Hayden Avenue
    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 Cuvitru
    D.2.1.1.2Name of the Marketing Authorisation holderBaxalta Innovations GmbH
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    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.3.4Pharmaceutical form Solution for injection
    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
    Primary Immunodeficiency Diseases (PID)
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of the study is to assess serum trough IgG concentrations following weekly administration of IGSC, 20% (Epoch 2) and serum trough IgG concentration after biweekly administration of IGSC, 20% (Epoch 3), in Japanese subjects with PID.
    E.2.2Secondary objectives of the trial
    - To assess serum trough IgG concentrations following every 3-week or every 4-week administration of IGIV (Epoch 1) in Japanese subjects with PID.
    - To characterize the PK profiles of IGSC, 20% in Japanese subjects with PID following weekly SC administration (Epoch 2).
    - To evaluate the safety and tolerability of IGSC, 20% (Epoch 2, Epoch 3) and of IGIV (Epoch 1) in Japanese subjects with PID.
    - To evaluate the efficacy of IGSC, 20% (Epoch 2, Epoch 3) and of IGIV (Epoch 1) in Japanese subjects with PID.
    - To assess quality of life aspects, treatment satisfaction, and treatment preference of Japanese subjects with PID (Epoch 1, Epoch 2, Epoch 3).
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be of Japanese descent, defined as born in Japan and having Japanese parents and Japanese maternal and paternal grandparents.
    2. Subject must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the IUIS Committee 2017 (Picard et al., 2018). The diagnosis must be confirmed by the Medical Director prior to treatment with IP.
    3. Subject is 2 years or older at the time of screening.
    4. Written informed consent is obtained from either the subject or the subject's legally authorized representative prior to any study-related procedures and study product
    administration.
    5. Subject has been receiving a consistent dose of IGIV over a period of at least 3 months prior to screening of 200 - 600 mg/kg BW at 3- or 4-week intervals.
    6. Subject has a serum trough level of IgG ≥5 g/L at screening.
    7. Subject has not had a serious bacterial infection within the 3 months prior to screening.
    8. Subject 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 amino transferase (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 presence of renal function impairment defined by estimated glomerular filtration rate [eGFR] <60 mL/min/1.73m2
    4. Subject has been diagnosed with or has a malignancy (other than adequately treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix), unless the
    disease-free period prior to screening exceeds 5 years.
    5. Subject is receiving anti-coagulation therapy or has a history of thrombotic episodes (including deep vein thrombosis, myocardial infarction, cerebrovascular accident,
    pulmonary embolism) within 12 months prior to screening or a history of thrombophilia.
    6. Subject has abnormal protein loss (protein losing enteropathy, nephrotic syndrome).
    7. Subject has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site.
    8. 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.
    9. Subject has immunoglobulin A (IgA) deficiency (IgA less than 0.07g/L), known anti IgA antibodies, and a history of hypersensitivity.
    10. Subject is on preventative (prophylactic) systemic antibacterial antibiotics at doses sufficient to treat or prevent bacterial infections, and cannot stop these antibiotics at the
    time of screening.
    11. Subject has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening.
    12. Subject has a bleeding disorder, or a platelet count less than 20,000/μL, or, in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result
    of subcutaneous therapy.
    13. Subject has total protein >9 g/dL or myeloma, or macroglobulinemia (IgM) or paraproteinemia.
    14. Women of childbearing potential meeting any one of the following criteria:
    a. Subject presents with a positive pregnancy test.
    b. Subject is breast feeding.
    c. Subject intends to begin nursing during the course of the study.
    d. Subject does not agree to employ adequate birth-control measures (e.g. intrauterine device, diaphragm or condom [for male partner] with spermicidal jelly or foam, or
    birth control pills/patches) throughout the course of the study.
    15. Subject has participated in another clinical study and has been exposed to an IP or device within 30 days prior to study enrollment.
    16. Subject is scheduled to participate in another non-observational (interventional) clinical study involving an IP or device during the course of the study.
    17. Subject has severe dermatitis that would preclude adequate sites for safe product administration.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the total serum trough levels of IgG (total serum trough IgG antibodies) measured during Period 2 of Study Epoch 2 (weekly administration of IGSC, 20%) and during Epoch 3 (biweekly administration of IGSC, 20%).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 26 weeks
    E.5.2Secondary end point(s)
    1. Epoch 1: Total Serum Trough Levels of Immune Globulin G (IgG) Antibodies
    2. Epoch 2: Area Under the Curve (AUC) for Total Serum Levels of IgG and IgG Subclasses
    3. Epoch 2: Apparent Clearance (CL/F) for Total Serum Levels of IgG and IgG Subclasses
    4. Epoch 2: Maximum Concentration (Cmax) for Total Serum Levels of IgG and IgG Subclasses
    5. Epoch 2: Minimum Concentration (Cmin) for Total Serum Levels of IgG and IgG Subclasses
    6. Epoch 2: Time to Maximum Concentration (Tmax) for Total Serum Levels of IgG and IgG Subclasses
    7. Trough Levels of Specific Antibodies to Clinically Relevant Pathogens
    8. Number of Participants with Treatment Emergent Adverse Events (TEAEs)
    9. Number of Participants with Tolerability Events Related to the Infusion of Investigational Product (IP)
    10. Annual Rate of Validated Acute Serious Bacterial Infections (ASBI)
    11. Annual Rate of All Infections
    12. Number of Days Participants not Able to Attend School or Work to Perform Normal Daily Activities due to Illness/Infection
    13. Number of Days Participants on Antibiotics
    14. Number of Participants Hospitalized due to Illness or Infection
    15. Length of Hospital Stay
    16. Number of Participants will Report Acute Physician Visits due to Illness/Infection
    17. Health-related Quality of Life (HRQoL): Pediatric Quality of Life Inventory (PedsQL)
    18. EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L)
    19. Health-related Quality of Life (HRQoL): Short Form-36 Health Survey (SF-36)
    20. Health Related Quality of Life: Treatment Satisfaction Questionnaire for Medication-9 (TSQM-9)
    21. Health Related Quality of Life: Treatment Satisfaction Questionnaire for Life Quality Index (LQI)
    22. Health Related Quality of Life: Treatment Preference
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to 57 weeks
    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
    Japan
    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 years1
    E.8.9.2In all countries concerned by the trial months4
    E.8.9.2In all countries concerned by the trial days11
    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: 6
    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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 11
    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 Yes
    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
    Pediatric subjects who couldn't personally give consent
    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 17
    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: Japan
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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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