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    Summary
    EudraCT Number:2022-004233-40
    Sponsor's Protocol Code Number:TAK-675-4008
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2026-07-01
    Trial 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 number2022-004233-40
    A.3Full title of the trial
    A Prospective, Open-label, Multicentre, Interventional, Single-arm, Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa [r-DNA Origin]) in Indian Children and Adults With Fabry Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Study of Replagal in Children and Adults With Fabry Disease in India
    A.4.1Sponsor's protocol code numberTAK-675-4008
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05067868
    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.1.3.4CountryUnited 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
    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 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-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 Replagal
    D.2.1.1.2Name of the Marketing Authorisation holderTakeda
    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 nameReplagal
    D.3.2Product code TAK-675
    D.3.4Pharmaceutical form Concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAgalsidase alfa
    D.3.9.1CAS number 104138-64-9
    D.3.9.2Current sponsor codeTAK-675
    D.3.9.3Other descriptive nameReplagal
    D.3.9.4EV Substance CodeSUB12456MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    Fabry Disease
    E.1.1.1Medical condition in easily understood language
    Fabry Disease
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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 main objective of the trial is to evaluate the safety of Replagal in Indian children and adults with Fabry disease during 53 weeks of study duration.
    E.2.2Secondary objectives of the trial
    • To evaluate the efficacy of Replagal in Indian children and adults with Fabry disease during 53 weeks of study duration.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male and female Replagal naïve participants (and who are not part of any other program that allows participant to get access to free enzyme replacement therapy (ERT) at the time of study enrollment and during the study period) of any age with confirmed diagnosis of Fabry disease.
    2. Participants who have documented confirmed diagnosis of Fabry disease based on proof of gene mutation: α-galactosidase A gene compatible with Fabry disease and/or a deficiency of α-galactosidase A (less than (<)4.0 nanomoles per milliliter per hour (nmol/mL/hour) in plasma or serum or <8% of average mean normal in leukocytes and sequencing of Alpha-galactosidase A (GLA) gene for females).
    3. Participant must have any clinical manifestations of Fabry disease based on investigator’s discretion.
    4. Participant/Legally Authorized Representative (LAR)/guardian is able to understand and willing to give written informed consent before performing any study specific procedures and willing to adhere to protocol requirements.
    5. Female participants of childbearing potential (e.g., nonsterilised, premenopausal female participants) must have a documented negative pregnancy test prior to administration of the first dose of Replagal in this study. In addition, all female participants of childbearing potential must use two medically accepted forms of contraception throughout the study, i.e., either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.
    6. Male participant who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (e.g., condom with or without spermicide) from signing of informed consent throughout the duration of the study.
    Note: Female participants not of childbearing potential defined as those who have been surgically sterilized (hysterectomy, bilateral oophorectomy, or tubal ligation) or who are postmenopausal (e.g., defined as at least 1 year since last regular menses with an appropriate clinical profile [i.e., age appropriate, history of vasomotor symptoms]).
    E.4Principal exclusion criteria
    1. Participants who have received Replagal.
    2. Participants with poorly controlled hypertension as per investigator’s discretion.
    3. Participants with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 milliliters per minute per 1.73 square meter (mL/min/1.73 m^2) and who had/will have kidney transplantation or are currently on dialysis.
    4. Participants with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either alanine aminotransferase (ALT) or aspartate aminotransferase (AST) level exceeded the value three times the upper limit of normal [ULN] and total bilirubin 1.5 times as high as the ULN); and deemed as clinically significant by investigstor for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the participant.
    5. If female, the participant is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or intending to donate ova during such time period.
    6. Participant/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
    7. Participant is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
    8. If male, the participant intends to donate sperm during the course of this study.
    9. Participants who had participated in any other investigational drug study within the past 4 weeks prior to screening.
    10. Any participant deemed as unfit for this trial, as per investigator’s clinical judgment.
    E.5 End points
    E.5.1Primary end point(s)
    - Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
    - Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)
    - Number of Participants With Adverse Drug Reactions (ADRs) Related to Replagal
    - Number of Participants With Infusion-related Reactions of Replagal
    E.5.1.1Timepoint(s) of evaluation of this end point
    From the study drug administration up to Week 53
    E.5.2Secondary end point(s)
    - Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)
    - Number of Participants With Change in Frequency and Regimen of Analgesic use of Replagal for Neuropathic Pain
    - Change From Baseline in Urine Concentration of Globotriaosylceramide (Gb3)
    - Change From Baseline in Urine Protein Creatinine Ratio
    - Percent Change From Baseline in Left Ventricular Mass Index (LVMI)
    - Percent Change From Baseline in Left Ventricular Wall Thickness
    - Percent Change From Baseline in Ejection Fraction
    - Change From Baseline in Quality of Life Based on Questionnaire 36-itme Form Survey (SF-36), Version 2, Acute (Physical and Mental Component Summary Scores)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline up to Week 53
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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
    India
    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
    Last visit of last participant
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    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: 1
    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) No
    F.1.1.5.1Number of subjects for this age range: 0
    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: 1
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    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 5
    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: India
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