Pegunigalsidase Alfa for Fabry Disease

Recruiting in Palo Alto (17 mi)

+30 other locations

Age: 18 - 65

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-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

Eligibility Criteria

This trial is for adult patients with Fabry Disease who completed previous studies (PB-102-F20, PB-102-F03, or PB-102-F30) and consent to participate. They must use effective contraception if they can have children. Those with conditions affecting study compliance are excluded.

Inclusion Criteria

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

You have already taken part in studies PB-102-F20, PB-102-F03, or PB-102-F30.

The patient signs informed consent

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.

Participant Groups

The trial tests the long-term safety and effectiveness of pegunigalsidase alfa at a dose of 1 mg/kg given intravenously every two weeks to adults with Fabry Disease who participated in earlier related trials.

1Treatment groups

Experimental Treatment

Group I: Experimental open labelExperimental Treatment1 Intervention

pegunigalsidase alfa