Pegunigalsidase Alfa for Fabry Disease
Trial Summary
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.
What data supports the effectiveness of the drug pegunigalsidase alfa for Fabry disease?
Research shows that pegunigalsidase alfa, a new enzyme replacement therapy, helps maintain kidney function and reduces harmful substances in the body for people with Fabry disease. It also has a longer-lasting effect in the bloodstream and causes fewer immune reactions compared to similar treatments.12345
Is pegunigalsidase alfa safe for humans?
How is the drug pegunigalsidase alfa different from other treatments for Fabry disease?
Pegunigalsidase alfa is unique because it is a PEGylated form of enzyme replacement therapy, which means it has a longer half-life in the body and is less likely to cause immune reactions compared to other treatments. This can lead to more stable and effective management of Fabry disease symptoms.12345
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
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive 1 mg/kg pegunigalsidase alfa intravenously every 2 weeks
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants continue receiving treatment to evaluate long-term safety and efficacy
Treatment Details
Interventions
- pegunigalsidase alfa
Find a Clinic Near You
Who Is Running the Clinical Trial?
Chiesi Farmaceutici S.p.A.
Lead Sponsor
Protalix
Lead Sponsor