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    Summary
    EudraCT Number:2022-004246-35
    Sponsor's Protocol Code Number:TAK-675-3001
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2024-06-10
    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 number2022-004246-35
    A.3Full title of the trial
    An Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of REPLAGAL® in Treatment-naïve Chinese Subjects 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 Treatment-naive Chinese Participants With Fabry Disease
    A.4.1Sponsor's protocol code numberTAK-675-3001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04974749
    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 Development Center Americas, 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 supportTakeda Development Center Americas, Inc
    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 Ave
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/00/002
    D.3 Description of the IMP
    D.3.1Product nameReplagal
    D.3.2Product code TAK-675
    D.3.4Pharmaceutical form Concentrate for solution for injection/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 study is to assess the safety of REPLAGAL (0.2 mg/kg every other week [EOW] up to 52 weeks), by evaluating the incidence of serious treatment-emergent adverse events (TEAEs) over the study period, in treatment-naïve Chinese subjects with Fabry disease.
    E.2.2Secondary objectives of the trial
    • To evaluate other safety parameters of REPLAGAL
    • To evaluate the efficacy of REPLAGAL on the renal parameter (ie. estimated glomerular filtration rate [eGFR])
    • To assess the efficacy of REPLAGAL on left ventricular mass index (LVMI) and left ventricular ejection fraction (LVEF)
    • To evaluate the efficacy of REPLAGAL on other renal variables, pain, and PD markers (plasma globotriaosylsphingosine [lyso-Gb3])
    • To evaluate the change in hearing for subjects <18 years old
    • To evaluate the PK of REPLAGAL in treatment-naïve Chinese subjects with Fabry disease.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subject and/or legally authorized representative must voluntarily sign an Institutional Review Board/Independent Ethics Committee approved 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 parent(s)/legally authorized representative should sign the ICF accordingly.
    2. The subject has confirmed diagnosis of Fabry disease as determined by the investigator, according to medical record including:
    • For male subject, Fabry disease is confirmed by a deficiency of GLA activity and a mutation in the GLA gene
    • For female subject, Fabry disease is confirmed by a mutation in the GLA gene.
    3. The subject is 7 to 65 years of age, inclusive, at screening.
    4. Female subjects of childbearing potential must have a negative pregnancy test at screening.
    5. Female subjects of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study and for at least 14 days after the final investigational product infusion
    6. The subject is deemed, as determined by the investigator, to have adequate general health to undergo the specified protocol-related procedures and to have no safety or medical contraindications for participation.
    7. The subject has not received any treatment (approved or investigational) specific to Fabry disease, such as ERT, chaperone therapy, or substrate reduction therapy.
    8. The adult subject (≥18 years old) must have an eGFR of 45 to 120 mL/min/1.73 m2 (inclusive). Serum creatinine is tested and the eGFR is calculated by central laboratory using the Chronic Kidney Disease Epidemiology (CKD-EPI) equation.
    E.4Principal exclusion criteria
    1. In the opinion of the investigator, the subject’s life expectancy is ≤5 years.
    2. The subject has undergone or is scheduled to undergo kidney transplantation or is currently on dialysis or has any signs or symptoms of end stage renal disease.
    3. The subject has a urine protein/creatinine ratio of >500 mg/g.
    4. The subject has a clinically relevant history of allergy or signs or symptoms of severe hypersensitivity, which in the investigator’s judgment, will substantially increase the subject’s risk if he or she participates in the study.
    5. In the opinion of the investigator, the subject has non-Fabry disease-related cause of end organ (renal, cardiovascular, central nervous system) dysfunction/failure or is receiving medications that may affect the rate of disease progression, as assessed by renal measures.
    6. The subject has a positive test result at screening for hepatitis B surface antigen with detectable hepatitis B viral DNA load, hepatitis C virus (HCV) antibody with confirmation by HCV ribonucleic acid polymerase chain reaction testing, or human immunodeficiency virus antibody.
    7. The subject has received prior treatment with any of the following medications, with the exception of non-systemic use:
    • Chloroquine, • Amiodarone, • Monobenzone, • Gentamicin
    8. The subject is pregnant or lactating.
    9. The subject has a body mass index >35 kg/m2.
    10. The subject is treated or has been treated with any investigational drug for indication other than Fabry disease within 30 days prior to study start.
    11. The subject and/or the subject’s parent(s)/legal guardian is unable to understand the nature, scope, and possible consequences of the study.
    12. The subject is unable to comply with the protocol, eg, uncooperative with protocol schedule, refusal to agree to all of the study procedures, inability to return for evaluations, or is otherwise unlikely to complete the study, as determined by the investigator.
    E.5 End points
    E.5.1Primary end point(s)
    Number of Participants With Serious Treatment-emergent Adverse Events (TEAEs)
    E.5.1.1Timepoint(s) of evaluation of this end point
    From start of study drug administration up to 14 days after end of treatment (EOT) period (up to Week 54)
    E.5.2Secondary end point(s)
    - Number of Participants With TEAEs
    - Number of Participants With Infusion-related Reactions (IRRs)
    - Number of Participants With Positive Anti-drug Antibody (ADA) to REPLAGAL
    - Number of Participants With Positive Neutralizing Antibody (NAb) to REPLAGAL
    - Number of Participants With Clinically Meaningful Changes in Laboratory Parameters
    - Number of Participants With Clinically Meaningful Changes in Vital Signs
    - Number of Participants With Clinically Meaningful Changes in 12-lead Electrocardiogram (ECG)
    - Change From Baseline in Renal Function at Week 52
    - Change From Baseline in Estimated Glomerular Filtration Rate (eGFR) Values at Weeks 8, 16, 28 and 40
    - Change From Baseline in Left Ventricular Mass Index (LVMI)
    - Change From Baseline in Left Ventricular Ejection Fraction (LVEF)
    - Change From Baseline in Urine Protein/Creatinine Ratio at Weeks 8, 16, 28, 40 and 52
    - Change From Baseline in Pain as Assessed by Brief Pain Inventory Short Form (BPI-short Form) at Weeks 8, 16, 28, 40 and 52
    - Change From Baseline in Plasma Globotriaosylsphingosine (Lyso-Gb3) Level at Weeks 8, 16, 28, 40 and 52
    - Change From Baseline in Audiology Testing Values
    - Area Under Serum Concentration-time Curve From the Time of Dosing to the Last Measurable concentration (AUC0-last) of REPLAGAL
    - Area Under Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf) of REPLAGAL
    - Serum Clearance of Administered Dose (CL) of REPLAGAL
    - Serum Clearance of Administered Dose Normalized Based on Body Weight of REPLAGAL
    - Maximum Observed Serum Concentration (Cmax) of REPLAGAL
    - Terminal Elimination Half-life (T1/2) of REPLAGAL
    - Time to Reach Maximum Observed Serum Concentration (Tmax) of REPLAGAL
    - Volume of Distribution at Steady State (Vss) of REPLAGAL
    - Volume of Distribution at Steady State Normalized Based on Body Weight of REPLAGAL
    - Dose Normalized Area Under Serum Concentration-time Curve From Time Zero to the Last Sampling Time (AUClast/Dose) of REPLAGAL
    - Dose Normalized Area Under the Serum Concentration-time Curve From Time Zero Extrapolated to Infinity (AUC0-inf/Dose) of REPLAGAL
    - Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of REPLAGAL
    E.5.2.1Timepoint(s) of evaluation of this end point
    From start of study drug administration up to 14 days after EOT period (up to Week 54)
    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 Yes
    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 months7
    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: 7
    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: 2
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 13
    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 No
    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 20
    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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