Study Summary
This trial will test if an infusible drug helps improve kidney function and heart structure in adults with Fabry Disease who have not had any prior treatment.
Treatment Effectiveness
Effectiveness Progress
Study Objectives
2 Primary · 10 Secondary · Reporting Duration: From start of study drug administration up to follow-up (Week 106)
Trial Safety
Safety Progress
Trial Design
1 Treatment Group
REPLAGAL
1 of 1
Experimental Treatment
45 Total Participants · 1 Treatment Group
Primary Treatment: REPLAGAL · No Placebo Group · Phase 3
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age 18 - 65 · All Participants · 9 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:Frequently Asked Questions
How many people are taking part in this research project at most?
"That is correct. The clinicaltrials.gov website indicates that the trial, which was created on December 28th 2021, is still recruiting patients. In total, 45 individuals are needed from 3 different locations." - Anonymous Online Contributor
How can I become involved in this experiment?
"This study is looking for 45 individuals that have been diagnosed with Fabry disease. The age range for participants is 18 to 65 years old. To be eligible, applicants must meet the following conditions: For male patients,Fabry disease must be confirmed by a deficiency of alpha-galactosidase A (GLA) activity and a mutation in the GLA gene. Participants must also voluntarily sign an Institutional Review Board (IRB)/Independent Ethics Committee/Research Ethics Board approved informed consent form after all relevant aspects of the study have been explained and discussed., Female patients of child-bearing potential who wish to participate in this" - Anonymous Online Contributor
Has the United States Food and Drug Administration cleared REPLAGAL for use?
"REPLAGAL's safety is estimated to be a 3. This Phase 3 clinical trial has supporting data for efficacy as well as multiple rounds of safety testing." - Anonymous Online Contributor
What are the precedence studies for this research?
"REPLAGAL has a long history, with the first trial being conducted in just 2021. Since then, there have been 3 additional trials held in 1 city and 22 countries." - Anonymous Online Contributor
Does this study still have room for more participants?
"That is correct, the online clinicaltrials.gov registry indicates that this study is currently recruiting patients. The trial was initially posted on December 28th, 2021 and was updated on August 8th, 2022. There are 45 spots available at 3 different locations." - Anonymous Online Contributor
Does this research require participants to be a certain age?
"This trial is only open to patients that fall between 18 and 65 years of age." - Anonymous Online Contributor
What are the specific goals of this research?
"The primary outcome of this trial is to assess the change in cardiac structure at Week 104. Secondary objectives include assessing the annualized rate of change in left ventricular mass index (LVMI) up to Week 104, the number of participants who will develop anti-drug antibodies (ADA) to REPLAGAL, and the annualized rate of change in estimated glomerular filtration rate (eGFR) up to Week 104." - Anonymous Online Contributor