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    Summary
    EudraCT Number:2023-000502-26
    Sponsor's Protocol Code Number:TAK-660-3001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2025-02-13
    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 number2023-000502-26
    A.3Full title of the trial
    A Phase 3, Prospective, Multicenter, Open-label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (ADYNOVATE) Administered for Prophylaxis and Treatment of Bleeding in Chinese Previously Treated Patients With Severe Hemophilia A (FVIII <1%)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Adynovate in Previously Treated Chinese Teenagers and Adults With Severe Hemophilia A
    A.4.1Sponsor's protocol code numberTAK-660-3001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05707351
    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 supportBaxalta US Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationTakeda Development Center Americas, Inc.
    B.5.2Functional name of contact pointStudy Director
    B.5.3 Address:
    B.5.3.1Street Address500 Kendall Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post codeMA 02142
    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 Adynovate/Adynovi
    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.3.4Pharmaceutical form Powder and solvent for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous 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) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    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
    Severe Hemophilia A (FVIII <1%)
    E.1.1.1Medical condition in easily understood language
    Hemophilia A
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    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 this study is to assess the efficacy of ADYNOVATE for prophylactic treatment in previously treated Chinese subjects with severe hemophilia A based on the total ABR.
    E.2.2Secondary objectives of the trial
    - To assess the efficacy of ADYNOVATE for prophylactic treatment based on ABR by bleeding site and cause
    - To assess the overall hemostatic efficacy rating of ADYNOVATE for treatment of nonsurgical breakthrough bleeding episodes during the study period
    - To assess the efficacy of ADYNOVATE for perioperative bleeding management if minor surgery is performed during the study period
    - To evaluate the safety of ADYNOVATE as assessed by AEs and serious AEs (SAEs) as well as clinically significant findings in vital signs and clinical laboratory parameters
    - To evaluate the safety and the immunogenicity of ADYNOVATE based on the incidence of FVIII inhibitory antibodies and binding antibodies to ADYNOVATE
    - To evaluate the PK of ADYNOVATE in Chinese subjects
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject and/or legally authorized representative must voluntarily sign a written informed consent form (ICF) after all relevant aspects of the study have been explained and discussed with the subject. For the subjects <18 years old, subjects will give assent AND their parents/legally authorized representative should sign the ICF accordingly.
    2. Subject and/or legally authorized representative understands and is willing and able to comply with all requirements of the study protocol.
    3. Subject should be ethnic Chinese.
    4. Subject is 12 to 65 years of age at screening and male.
    5. Subject has severe hemophilia A (FVIII clotting activity <1%) as confirmed by the central laboratory at screening after a washout period of at least 72 to 96 hours.
    6. The last on-demand or prophylactic treatment received is within 3 months before screening.
    7. Subject has documented previous treatment with plasma-derived FVIII concentrates or recombinant FVIII for >150 EDs.
    8. Subject is human immunodeficiency virus (HIV)-negative, or HIV-positive with stable disease and CD4+ count ≥200 cells/mm3.
    9. Subject is hepatitis C virus (HCV) negative by antibody testing (if positive, additional polymerase chain reaction testing will be performed to confirm), as confirmed at screening; or HCV-positive with chronic stable hepatitis, as assessed by the investigator.
    E.4Principal exclusion criteria
    1. Subject has detectable FVIII inhibitory antibodies (≥0.6 Bethesda units (BU)/mL using the Nijmegen modification of the Bethesda assay) as confirmed by the central laboratory at screening.
    2. Subject has a confirmed history of FVIII inhibitory antibodies (≥0.6 BU using the Nijmegen modification of the Bethesda assay or ≥0.6 BU using the Bethesda assay) at any time prior to screening.
    3. Subject has a known hypersensitivity to ADYNOVATE or ADVATE or any of the components of the study drugs, such as mouse or hamster proteins, or other FVIII products.
    4. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease).
    5. Subject has severe hepatic dysfunction (eg, ≥5 times the upper limit of normal [ULN] for alanine aminotransferase (ALT) or aspartate aminotransferase (AST), a recent or persistent international normalized ratio [INR] >1.5, as confirmed by the local laboratory at screening).
    6. Subject has severe renal impairment (serum creatinine >1.5 times the ULN) as confirmed by the local laboratory at screening.
    7. Subject is planned or likely to undergo major surgery during the study period.
    8. Subject has current or recent (<30 days) use of other PEGylated drugs before study participation or scheduled use of such drugs during study participation.
    9. Subject has received emicizumab therapy within 6 months of screening.
    10. Subject is currently receiving, or scheduled to receive during the study, an immunomodulating drug (eg, systemic corticosteroid agent at a dose equivalent to hydrocortisone >10 mg/day, or α-interferon) other than antiretroviral chemotherapy.
    11. Subject has participated in another clinical study involving the use of an IP other than ADYNOVATE or an investigational device within 30 days before the screening visit or is scheduled to participate in another clinical study involving an IP or investigational device during this study.
    12. Subject has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance.
    13. Subject, in the opinion of the investigator, is unable or unwilling to comply with the study protocol.
    E.5 End points
    E.5.1Primary end point(s)
    Total Annualized Bleeding Rates (ABR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline up to a minimum of 50 exposure days(EDs) to ADYNOVATE or approximately 26 weeks (+2 weeks), whichever occurs last
    E.5.2Secondary end point(s)
    - ABR Based on Bleeding Site and Cause
    - Number of Adynovate Infusions per Week During the Prophylactic Treatment Period
    - Number of Adynovate Infusions per Month During the Prophylactic Treatment Period
    - Weight-adjusted Consumption of Adynovate per Week During the Prophylactic Treatment Period
    - Weight-adjusted Consumption of Adynovate per Month During the Prophylactic Treatment Period
    - Percentage of Participants With Zero Bleeding Episodes During the Study
    - Time Intervals Between Bleeding Episodes
    - Overall Hemostatic Efficacy Rating at Bleed Resolution for Treatment of Breakthrough Bleeding Episodes
    - Number of Adynovate Infusions per Bleeding Episode
    - Weight-adjusted Consumption of Adynovate per Bleeding Episode
    - Number of Minor Surgeries With Hemostatic Efficacy Response Based on Global Hemostatic Efficacy Assessment (GHEA) Score as Assessed by the Operating Surgeon/Investigator
    - Volume of Actual and Predicted Intra-operative and Post-operative Blood Loss After the Surgery as Assessed by the Operating Surgeon/Investigator
    - Number of Participants who Require Perioperative Transfusion of Blood, Red blood Cells, Platelets, and Other Blood Products
    - Daily Intra-Operative and Post-Operative Weight-Adjusted Consumption Dose of Adynovate
    - Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
    - Number of Participants With Confirmed Inhibitory Antibodies to Factor VIII (FVIII), Binding Antibodies to Adynovate and Chinese Hamster Ovary (CHO) Protein
    - FVIII Activity Level in Plasma Assessed by a 1-stage Clotting Assay
    - Incremental Recovery Over Time During Adynovate Prophylactic Treatment
    - Pre-dose Level of FVIII Activity and Antigen and von Willebrand Factor (VWF) Antigen in Plasma
    - Clearance (CL) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    - Volume of Distribution (V) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    - Area Under the Concentration Versus Time Curve From 0 to 96 Hours (AUC0-96) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    - Maximum Concentration (Cmax) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    - Pre-dose Concentration (Cpredose) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    - Terminal Phase Elimination Half-life (T1/2) for FVIII Activity Following an Initial Single Dose and Steady-state Dose of Adynovate
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline up to a minimum of 50 exposure days(EDs) to ADYNOVATE or approximately 26 weeks (+2 weeks), whichever occurs last
    PK endpoints: Baseline and Week 20
    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.8.2.4Number of treatment arms in the trial1
    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
    China
    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 months6
    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: 10
    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: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 27
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    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 No
    F.3.3.7Others Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Minors
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 37
    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: China
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