NCT05067868 A Study of Replagal in Children and Adults With Fabry Disease in India
| NCT ID | NCT05067868 |
| Status | Recruiting |
| Phase | Phase 4 |
| Sponsor | Shire |
| Condition | Fabry Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 5 participants |
| Start Date | 2022-11-01 |
| Primary Completion | 2026-10-31 |
Trial Parameters
Eligibility Fast-Check
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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.
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