Replagal for children and adults with Fabry disease in India

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

The study will try giving Replagal every two weeks for about a year to children and adults in India who have Fabry disease to learn more about its safety.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sex: All
• Sponsor: Takeda (Industry-sponsored)
• Locations: 3 sites (Kolkata and 2 other locations)
• Trial ID: NCT05067868 on ClinicalTrials.gov

What this trial studies

This Phase 4 interventional study enrolls Replagal‑naïve participants of any age with genetically or enzymatically confirmed Fabry disease and clinical manifestations. Participants receive Replagal infusions at the clinic every two weeks for approximately one year. The primary aim is to characterize the safety profile of Replagal in this population with regular follow‑up during the treatment period. Study sites include major centers in Kolkata and New Delhi and require written informed consent from participants or their legal representatives.

Who should consider this trial

Good fit: Ideal candidates are people of any age in India with a confirmed GLA gene mutation or enzyme deficiency consistent with Fabry disease, who have not previously received Replagal or enrolled in another free ERT program and who can provide informed consent or have a legal representative do so.

Not a fit: Patients already receiving enzyme replacement therapy, those without a confirmed diagnosis of Fabry disease, or those unable to attend biweekly clinic visits are unlikely to benefit from joining this study.

Why it matters

Potential benefit: If successful, the study could provide additional safety data to support use of Replagal and help expand access to enzyme replacement therapy in India.

How similar studies have performed: Replagal (agalsidase alfa) has been used in other studies and clinical practice with demonstrated benefits and an established safety profile, and this study aims to add region‑specific safety data.

Eligibility criteria

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 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).
3. Participant must have any clinical manifestations of Fabry disease based on investigator's discretion.
4. 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.
5. 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.
6. 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 \[that is, age appropriate, history of vasomotor symptoms\]).

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 milliliter per minute (mL/min) /1.73 meter square (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 investigator 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, 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.
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.

Where this trial is running

Kolkata and 2 other locations

• Institute of Child Health — Kolkata, India (Not_yet_recruiting)
• All India Institute of Medical Sciences (AIIMS) — New Delhi, India (Recruiting)
• Sir Gangaram Hospital — New Delhi, India (Recruiting)

Study contacts

• Study coordinator: Takeda Contact

Conditions Fabry Disease