A Study of Replagal in Children and Adults With Fabry Disease in India

NCT05067868 | Recruiting Phase 4

• 2 other identifiers: TAK-675-40082022-004233-40
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 3

Brief Summary

The main aim of this study is to learn more about the safety profile of Replagal. Participants will receive Replagal every 2 weeks at the clinic for about 1 year.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical (study) product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal (study) product, whether or not related to the medicinal (study) product. An SAE is any untoward medical occurrence (whether considered to be related to study product or not) that at any dose results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality or birth defect, an important medical event.

  From the start of study up to 53 weeks

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Treatment Emergent Serious Adverse Events (TESAEs)

  AE is any unfavorable and unintended sign, symptom, or disease temporally associated with study or use of investigational drug product (IP), whether or not the AE is considered related to IP. TEAEs: AEs occurring or worsening at or after first dose of IP or ongoing at time of enrollment. SAE :untoward medical occurrence that at any dose met one, more of the following criteria: results in death, life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent, significant disability/incapacity, a congenital abnormality/birth defect, an important medical event. Severity: Mild: event that does not generally interfere with usual activities of daily living; Moderate: event that interferes with usual activities of daily living, causing discomfort, permanent risk of harm; Severe: AE that interrupts usual activities of daily living, significantly affects clinical status, or may require intensive therapeutic intervention.

  From the study drug administration up to Week 53

• Number of Participants With Adverse Drug Reactions (ADRs) Related to Replagal

  An ADR is defined as a response to a drug which is noxious and unintended, and which occurs at doses normally used in humans for prophylaxis, diagnosis, or therapy of disease, or for the modification of physiological function. Number of participants with ADRs will be reported.

  From the study drug administration up to Week 53

• Number of Participants With Infusion-related Reactions of Replagal

  Number of participants with infusion-related reactions of Replagal will be reported.

  From the study drug administration up to Week 53

Secondary Outcomes (8)

• Change From Baseline in Estimated Glomerular Filtration Rate (eGFR)

  Baseline and at Weeks 13, 27, 39, and 53

• Number of Participants With Change in Frequency and Regimen of Analgesic use of Replagal for Neuropathic Pain

  Baseline up to Week 53

• Change From Baseline in Urine Concentration of Globotriaosylceramide (Gb3)

  Baseline and at Weeks 13, 27, 39, and 53

• Change From Baseline in Urine Protein Creatinine Ratio

  Baseline and at Weeks 13, 27, 39, and 53

• Percent Change From Baseline in Left Ventricular Mass Index (LVMI)

  Baseline and at Weeks 27 and 53

Study Arms (1)

Replagal

EXPERIMENTAL

Participants with fabry disease will receive Replagal 0.2 milligram per kilogram (mg/kg) intravenous infusion on Day 1 and every 2 weeks up to Week 51.

Biological: Replagal

Interventions

Replagal BIOLOGICAL

Participants will receive Replagal 0.2 mg/kg, intravenous infusion at Day 1 and every 2 weeks.

Also known as: Agalsidase alfa

Replagal

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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 nanomole per milliliter per hour (nmol/mL/hour) in plasma or serum or \<8 percent (%) of average mean normal in leukocytes and sequencing of GLA gene for females).
• Participant must have any clinical manifestations of Fabry disease based on investigator's discretion.
• Participant/legal 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.
• Female participants of childbearing potential (example, 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 a two medically accepted forms of contraception throughout the study, that is, either a barrier method or hormonal contraceptive with norethindrone and ethinyl estradiol or similar active components.
• Male participant who is nonsterilised and sexually active with a female partner of childbearing potential agrees to use barrier method of contraception (example, 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 (example, defined as at least 1 year since last regular menses with an appropriate clinical profile \[that is, age appropriate, history of vasomotor symptoms\]).

You may not qualify if:

• Participants who have received Replagal.
• Participants with poorly controlled hypertension as per investigator's discretion.
• Participants with chronic kidney disease (CKD) with estimated Glomerular Filtration rate less than 15 milliliter per minute (mL/min) /1.73 meter square (m\^2) and who had/will have kidney transplantation or are currently on dialysis.
• 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 investigator for hematology and biochemistry. These abnormal laboratory values could be discussed with medical monitor before excluding the participant.
• 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.
• Participant/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
• Participant is unable to comply with the protocol, example, 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.
• If male, the participant intends to donate sperm during the course of this study.
• Participants who had participated in any other investigational drug study within the past 4 weeks prior to screening.
• Any participant deemed as unfit for this trial, as per investigator's clinical judgment.

Sponsors & Collaborators

• Shire: lead
• Takeda: collaborator

Study Sites (3)

Institute of Child Health

Kolkata, 700017, India

NOT YET RECRUITING

All India Institute of Medical Sciences (AIIMS)

New Delhi, 110029, India

RECRUITING

Sir Gangaram Hospital

New Delhi, 110060, India

RECRUITING

Related Links

• To obtain more information on the study, click here/on this link

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase alfa

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1 866 842 5335; ClinicalTransparency@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 24, 2021
• First Posted: October 5, 2021
• Study Start: November 1, 2022
• Primary Completion (Estimated): October 31, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: September 19, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/ For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

Locations

IN (3)