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New treatment option for Fabry Disease
Official title A Study of Replagal in Children and Adults With Fabry Disease in India
ClinicalTrials.gov ID: NCT05067868
What this study is testing
What is Replagal?
Replagal is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Also referred to as Agalsidase alfa.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 4: studies an already-approved treatment
- Time commitment: about 1 year
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults
You may be able to join 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...
- Participants who have documented confirmed diagnosis of Fabry disease based on proof of gene mutation: α-galactosidase A gene compatible with Fabry...
- 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...
- Female participants of childbearing potential (example, nonsterilised, premenopausal female participants) must have a documented negative pregnancy...
You likely can't join 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...
- Participants with any serious hepatic disorder who had abnormal hepatic function test values at screening (when either alanine aminotransferase [ALT]...
- If female, the participant is pregnant or lactating or intending to become pregnant before participating in this study, during the study; or...
- Participant/LAR/guardian is unable to understand the nature, scope, and possible consequences of the study.
See the full eligibility criteria
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Kolkata, India
- New Delhi, India
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 1 year per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Kolkata, India; New Delhi, India. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.