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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
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from start of study drug administration up to follow-up visit (i.e., up to week 106)
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial will treat adults with untreated Fabry Disease using Replagal, an infusion medication. The goal is to see if it improves kidney function and heart structure by replacing a missing enzyme to reduce fat buildup. Replagal (agalsidase alfa) is an enzyme replacement therapy approved in Europe for treating Fabry disease.

Eligible Conditions
  • Fabry Disease

Timeline

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change From Baseline in Cardiac Structure at Week 104
Change From Baseline in Renal Function at Week 104
Secondary study objectives
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

Side effects data

From 2024 Phase 3 trial • 20 Patients • NCT04974749
65%
COVID-19
30%
Cough
25%
Pyrexia
25%
Upper respiratory tract infection
15%
Headache
10%
Corneal opacity
10%
Oedema peripheral
10%
Angina pectoris
10%
Albuminuria
10%
Back pain
10%
Chest discomfort
10%
Bone metabolism disorder
10%
Haematuria
5%
Sepsis
5%
Ectopic pregnancy
100%
80%
60%
40%
20%
0%
Study treatment Arm
REPLAGAL

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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
96,013 Total Patients Enrolled
12 Trials studying Fabry Disease
4,479 Patients Enrolled for Fabry Disease
Study DirectorStudy DirectorShire
1,276 Previous Clinical Trials
499,261 Total Patients Enrolled
14 Trials studying Fabry Disease
13,537 Patients Enrolled for Fabry Disease
~4 spots leftby Nov 2025
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