Pegunigalsidase Alfa for Fabry Disease
(Bright51 Trial)

Recruiting in Palo Alto (17 mi)

+16 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Chiesi Farmaceutici S.p.A.

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Research Team

Eligibility Criteria

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.

Inclusion Criteria

Completion of study PB-102-F50

The patient signs informed consent

I agree to use effective birth control during and for 2 weeks after the treatment.

Exclusion Criteria

Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.

Treatment Details

Interventions

pegunigalsidase alfa (Enzyme Replacement Therapy)

Trial OverviewThe 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.

Participant Groups

1Treatment groups

Experimental Treatment

Group I: Experimental open labelExperimental Treatment1 Intervention

Pegunigalsidase alfa