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Enzyme Replacement Therapy

Pegunigalsidase Alfa for Fabry Disease (Bright51 Trial)

Phase 3
Waitlist Available
Research Sponsored by Chiesi Farmaceutici S.p.A.
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 once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
Awards & highlights
No Placebo-Only Group
Pivotal Trial

Summary

This trial is testing how safe and effective a drug is in treating Fabry disease in adults who have completed another trial successfully.

Who is the study for?
This trial is for adult Fabry disease patients who completed the PB-102-F50 study. Participants must agree to use effective contraception if they or their partners can have children, and sign an informed consent. Those with conditions that could affect study compliance are excluded.
What is being tested?
The trial tests the long-term safety and effectiveness of a drug called pegunigalsidase alfa, given intravenously at a dose of 2 mg/kg every four weeks to adults with Fabry disease who previously finished another related trial.
What are the potential side effects?
While specific side effects are not listed here, common ones for similar enzyme replacement therapies include allergic reactions, infusion-related reactions, pain in extremities, headache, fatigue, nausea, fever and chills.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
This trial's timeline: 3 weeks for screening, Varies for treatment, and once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Evaluation of treatment-related adverse events
Secondary study objectives
Biomarkers for Fabry disease
Cardiac assessment
Clinical assessment
+5 more

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: Experimental open labelExperimental Treatment1 Intervention
Pegunigalsidase alfa

Find a Location

Who is running the clinical trial?

Chiesi Farmaceutici S.p.A.Lead Sponsor
202 Previous Clinical Trials
313,000 Total Patients Enrolled
11 Trials studying Fabry Disease
423 Patients Enrolled for Fabry Disease
ProtalixLead Sponsor
18 Previous Clinical Trials
460 Total Patients Enrolled
9 Trials studying Fabry Disease
283 Patients Enrolled for Fabry Disease

Media Library

pegunigalsidase alfa (Enzyme Replacement Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03614234 — Phase 3
Fabry Disease Research Study Groups: Experimental open label
Fabry Disease Clinical Trial 2023: pegunigalsidase alfa Highlights & Side Effects. Trial Name: NCT03614234 — Phase 3
pegunigalsidase alfa (Enzyme Replacement Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03614234 — Phase 3
~4 spots leftby Dec 2025
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