Actively Recruiting
A Phase IV Study to Evaluate the Safety and Efficacy of Replagal (Agalsidase Alfa) in Indian Children and Adults With Fabry Disease
Led by Shire · Updated on 2025-09-19
5
Participants Needed
3
Research Sites
4 weeks
Total Duration
On this page
Sponsors
S
Shire
Lead Sponsor
T
Takeda
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and effectiveness of Replagal in children and adults with Fabry disease in India. This open-label, single-arm Phase IV study aims to better understand the safety profile of Replagal by monitoring participants for about one year. Fabry disease participants must have a confirmed diagnosis based on gene mutation or enzyme deficiency and show clinical symptoms. Participants will receive Replagal at a dose of 0.2 mg per kilogram through intravenous infusion starting on Day 1 and then every two weeks up to Week 51. The treatment is given at the clinic and is designed to assess safety and potential benefits over approximately one year. This study does not include a comparison group and focuses on the effects of Replagal alone. During the study, participants will have regular assessments including kidney function tests, heart measurements, urine analyses, and quality of life questionnaires at various time points up to Week 53. Researchers will monitor adverse events, infusion reactions, and any treatment-related side effects throughout the study. Participants will be followed closely with scheduled visits to evaluate safety and any changes in their condition over the course of the year.
CONDITIONS
Brief Title
A Study of Replagal in Children and Adults With Fabry Disease in India
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male and female participants of any age with confirmed Fabry disease who have not previously received Replagal
- Diagnosis confirmed by gene mutation or enzyme deficiency tests
- Participants with clinical symptoms of Fabry disease as determined by the investigator
- Able to understand and willing to provide written informed consent
- Females of childbearing potential must have a negative pregnancy test and agree to use two forms of contraception during the study
- Nonsterilized, sexually active male participants agree to use barrier contraception throughout the study
You will not qualify if you...
- Participants who have already received Replagal
- Poorly controlled hypertension as judged by the investigator
- Chronic kidney disease with very low kidney function (eGFR less than 15 mL/min/1.73 m2), kidney transplant patients, or those on dialysis
- Serious liver disorders with abnormal liver test results deemed significant by the investigator
- Pregnant or lactating females or those intending pregnancy or egg donation during the study
- Unable to understand the study or comply with protocol procedures
- Male participants intending to donate sperm during the study
- Participation in any other investigational drug study within 4 weeks prior to screening
- Any other condition considered unsuitable by the investigator based on clinical judgment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 51 weeks
Participants receive Replagal 0.2 mg/kg by intravenous infusion on Day 1 and every 2 weeks up to Week 51.
Approximately 26 infusions given every 2 weeks
Duration - Up to 2 weeks after last treatment
Participants are monitored for safety and efficacy outcomes up to Week 53 after treatment begins.
Visits at Weeks 52 and 53 for assessments
Trial Site Locations
Total: 3 locations
1
Institute of Child Health
Kolkata, India, 700017
Not Yet Recruiting
2
All India Institute of Medical Sciences (AIIMS)
New Delhi, India, 110029
Actively Recruiting
3
Sir Gangaram Hospital
New Delhi, India, 110060
Actively Recruiting
Research Team
T
Takeda Contact
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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