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    Summary
    EudraCT Number:2022-003622-45
    Sponsor's Protocol Code Number:TAK-771-3004
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2022-12-06
    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 number2022-003622-45
    A.3Full title of the trial
    A Phase 3, Open-label, Non-controlled Study to Evaluate the Pharmacokinetics, Safety and Tolerability, and Efficacy of TAK-771 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 TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID)
    A.4.1Sponsor's protocol code numberTAK-771-3004
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05150340
    A.5.4Other Identifiers
    Name:jRCTNumber:jRCT2031210457
    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 SponsorTakeda
    B.1.3.4CountryJapan
    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
    B.4.2CountryJapan
    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 pointStudy Director
    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-mailTrialDisclosures@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 holderTakeda
    D.2.1.2Country which granted the Marketing AuthorisationJapan
    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.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHuman normal immunoglobulin
    D.3.9.1CAS number 308067-58-5
    D.3.9.2Current sponsor codeTAK-771
    D.3.9.3Other descriptive nameHUMAN NORMAL IMMUNOGLOBULIN
    D.3.9.4EV Substance CodeSUB14196MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10064859
    E.1.2Term Primary immunodeficiency syndrome
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess serum trough levels of total immunoglobulin G (IgG) when using TAK-771 as maintenance therapy in Japanese participants with PID.
    E.2.2Secondary objectives of the trial
    - To characterize the pharmacokinetic (PK) profiles of TAK-771 in Japanese participants with PID following TAK-771 administration.
    - To evaluate the safety and tolerability of TAK-771 in Japanese participants with PID.
    - To evaluate the efficacy of TAK-771 in Japanese participants with PID.
    - To assess disease activity and health-related quality of life (HRQoL) in Japanese participants with PID following TAK-771 administration.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Be a Japanese person.
    2. Participant must have a documented diagnosis of a form of primary humoral immunodeficiency involving antibody formation and requiring gammaglobulin replacement, as defined according to the International Union of Immunological Societies(IUIS) Committee 2017. The diagnosis must be confirmed by the Medical Director prior to TAK-771 treatment.
    3. Participant has been receiving a stable clinical dose of intravenous immunoglobulin (IVIG) or conventional subcutaneous immunoglobulin (cSCIG), which is equivalent to approximately 200 to 600 mg/kg body weight per 3 to 4 week period for IVIG and approximately 50 to 200 milligrams per kilogram (mg/kg) body weight per week for cSCIG based on the description in the package insert, consistently over a period of at least 3 months prior to screening, or Participant has been receiving of TAK-664 with fixed dose and dosing frequency at least 3 months prior to enrollment. That is, participant is about to complete Study TAK-664-3001 or participating in Study TAK-664-3002.
    4. Participant who has been receiving IVIG or cSCIG had all serum trough levels of total IgG ≥5 grams per liter (g/L) within 1 month prior to the screening/enrollment.
    5. Serum trough levels at screening/enrollment meet one of the following:
    a. IVIG-treated or cSCIG-treated participants Participant who had serum trough levels of IgG ≥5 g/L at the last 2 points in screening procedure before the first administration of TAK-771.
    b. TAK-664-treated participants Participant who had serum trough levels of IgG ≥5 g/L at the last 2 points in TAK-664 studies before the first administration of TAK-771.
    6. Participant is willing and able to comply with use of digital tools and applications.
    E.4Principal exclusion criteria
    1. Participant has a known history of or is positive at screening/enrollment 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 For participants who are switching from TAK-664 studies, the eligibility will be reconfirmed after result of the specialty test conducted at Week 1 become available.
    2. Abnormal laboratory values at screening/enrollment 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/millimeter (mm)^3)
    3. Participant has presence of renal function impairment defined by eGFR <60 milliliters (mL)/ minute (min)/1.73 meter (m)^2.
    4. Participant has been diagnosed with, or had 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. Participant 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/enrollment or a history of thrombophilia.
    6. Participant has abnormal protein loss (protein losing enteropathy, nephrotic syndrome).
    7. Participant has anemia that would preclude phlebotomy for laboratory studies according to standard practice at the site.
    8. Participant has an ongoing history of hypersensitivity or persistent reactions (urticaria, breathing difficulty, severe hypotension, or anaphylaxis) following IVIG, subcutaneous immunoglobulin (SCIG), and/or Immune Serum Globulin infusions
    9. Participant has immunoglobulin A (IgA) deficiency (serum IgA less than 0.07g/L) and history of hypersensitivity, or history of confirmed anti-IgA antibodies, or both.
    10. Participant 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/enrollment.
    11. Participant has active infection and is receiving antibiotic therapy for the treatment of infection at the time of screening/enrollment or had a serious bacterial infection within the 3 months prior to screening/enrollment
    12. Participant has a bleeding disorder, or a platelet count less than 20,000/microliters (microL), or in the opinion of the investigator, would be at significant risk of increased bleeding or bruising as a result of subcutaneous (SC) therapy.
    13. Participant has total protein >9 grams per deciliter (g/dL) or myeloma, or macroglobulinemia (IgM) or paraproteinemia.
    14. Participant has a known allergy to hyaluronidase
    15. Participant has severe dermatitis that would preclude adequate sites for safe product administration.
    E.5 End points
    E.5.1Primary end point(s)
    Epoch 2: Serum Trough Levels of Total IgG Antibodies after Administration of TAK-771
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
    E.5.2Secondary end point(s)
    1. Epoch 2: Maximum Concentration (Cmax) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    2. Epoch 2: Time to Maximum Concentration (Tmax) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    3. Epoch 2: Area Under the Curve (AUC) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    4. Epoch 2: Half-life of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    5. Epoch 2: Apparent Total Clearance (CL/F) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    6. Epoch 2: Apparent Volume of Distribution (Vz/F) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    7. Epoch 2: Minimum Concentration (Cmin) of Total Serum Levels of IgG and IgG subclasses (IgG1, IgG2, IgG3, and IgG4)
    8. Epoch 2: Serum Trough Levels of IgG subclasses (IgG1, IgG2, IgG3, and IgG4) after Administration of TAK-771
    9. Epoch 1: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens after Administration of TAK-771
    10. Epoch 2: Trough Levels of Specific Antibodies to Clinically Relevant Pathogens after Administration of TAK-771
    11. Percentage of Participants with Treatment-Emergent Adverse Events (TEAEs)
    12. Percentage of Participants with TAK-771-related and TAK-771-non-related TEAEs
    13. Percentage of Participants with Serious and Non-serious TEAEs
    14. Percentage of Participants with Severe TEAEs
    15. Percentage of Participants with Local and Systemic TEAEs
    16. Percentage of Participants with TEAEs Leading to Premature Discontinuation from Study
    17. Percentage of Participants with Infusion-associated TEAEs
    18. Percentage of Participants with Clinically Significant Changes in Clinical Laboratory Parameters Recorded as TEAEs
    19. Percentage of Participants with Clinically Significant Changes in Vital Signs and Body Weight Recorded as TEAEs
    20. Epoch 2: Percentage of Participants who Develop Anti-rHuPH20 Binding Antibody Titers of Greater Than or Equal to 1:160
    21. Epoch 2: Percentage of Participants who Develop Neutralizing Antibodies to rHuPH20
    22. Percentage of Participants who Experienced Tolerability Events Related to the Infusion of TAK-771
    23. Epoch 1: Number of Weeks to Reach Final Dose Interval (3 Weeks or 4 Weeks)
    24. Epoch 2: Percentage of Participants who Achieve a Treatment Interval of 3 or 4 Weeks
    25. Epoch 2: Percentage of Participants who Maintain a Treatment Interval of 3 or 4 Weeks
    26. Annual Rate of Validated Acute Serious Bacterial Infections (ASBIs)
    27. Annual Rate of All Infections per Participant
    28. Healthcare Resource Utilization: Days Not Able To Attend School/Work or To Perform Normal Daily Activities Due to Illness/Infection
    29. Healthcare Resource Utilization: Days on Antibiotics
    30. Healthcare Resource Utilization: Number of Hospitalizations Due to Illness/Infection
    31. Healthcare Resource Utilization: Length of Stay in Days of Hospitalizations Due to Illness/Infection
    32. Healthcare Resource Utilization: Number of Acute (Urgent or Unscheduled) Physician Visits Due to Illness/Infection
    33. Infusion Parameters in Epoch 2: Number of Infusions per Month
    34. Infusion Parameters in Epoch 2: Number of Infusion Sites per Infusion
    35. Infusion Parameters in Epoch 2: Number of Infusion Sites per Month
    36. Infusion Parameters in Epoch 2: Duration of Individual Infusions
    37. Infusion Parameters in Epoch 2: Maximum Infusion Rate per Site
    38. Infusion Parameters in Epoch 2: Infusion Volume per Site
    39. Quality of Life (QOL): Pediatric Quality of Life Inventory (PEDS-QL)
    40. QOL: Short Form-36 Health Survey Version 2 (SF-36 v2)
    41. QOL: EuroQoL (Quality of Life)-5 Dimensions 3 Levels (EQ-5D-3L) Health Questionnaire
    42. Treatment Satisfaction: Questionnaire for Medication-9 (TSQM-9)
    43. Treatment Preference
    E.5.2.1Timepoint(s) of evaluation of this end point
    Up to Week 31 for Participants with 4-Week Dosing Interval or Up to Week 28 for Participants with 3-Week Dosing Interval
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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.8.2.4Number of treatment arms in the trial2
    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 months3
    E.8.9.2In all countries concerned by the trial days7
    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: 2
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 1
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 1
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 7
    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
    Being pediatric age group, participants were not able to give consent, thus needed parent/caregiver to sign 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 15
    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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