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Enzyme Replacement Therapy

REPLAGAL for Fabry Disease

Phase 3
Waitlist Available
Research Sponsored by Shire
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
The participant has not received any treatment (approved or investigational) specific to Fabry disease, such as enzyme replacement therapy (ERT), chaperone therapy, or substrate reduction therapy.
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from start of study drug administration up to follow-up (week 106)
Awards & highlights

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.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have not received any treatment for Fabry disease, including approved or experimental medication like enzyme replacement therapy, chaperone therapy, or substrate reduction therapy.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from start of study drug administration up to follow-up (week 106)
This trial's timeline: 3 weeks for screening, Varies for treatment, and from start of study drug administration up to follow-up (week 106) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in Cardiac Structure at Week 104
Change From Baseline in Renal Function at Week 104
Secondary outcome measures
Annualized Rate of Change in Estimated Glomerular Filtration Rate (eGFR) up to Week 104
Annualized Rate of Change in Left Ventricular Mass Index (LVMI) up to Week 104
Change From Baseline in Cardiac Fibrotic Segments up to Week 104
+7 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: REPLAGALExperimental Treatment1 Intervention
Participants will receive REPLAGAL 0.2 milligram per kilogram (mg/kg) body weight of intravenous (IV) infusion Every Other Week (EOW) for 104 weeks.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
REPLAGAL
2022
Completed Phase 3
~20

Find a Location

Who is running the clinical trial?

ShireLead Sponsor
456 Previous Clinical Trials
97,275 Total Patients Enrolled
12 Trials studying Fabry Disease
4,479 Patients Enrolled for Fabry Disease
Study DirectorStudy DirectorShire
1,202 Previous Clinical Trials
489,615 Total Patients Enrolled
14 Trials studying Fabry Disease
13,537 Patients Enrolled for Fabry Disease

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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."

Answered by AI

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"

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI

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."

Answered by AI
~5 spots leftby Mar 2025