Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
1 other identifier
expanded_access
N/A
1 country
11
Brief Summary
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.
Trial Health
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11 active sites
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Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 11, 2020
CompletedFirst Posted
Study publicly available on registry
September 17, 2020
CompletedJuly 31, 2024
July 1, 2024
September 11, 2020
July 30, 2024
Conditions
Keywords
Interventions
Pegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks).
Eligibility Criteria
You may qualify if:
- In the opinion of the Treating Physician, the patient cannot be adequately treated with any FDA approved drugs for Fabry and is not able to enroll in any current clinical trial for Fabry disease.
- Patient (or legal guardian) is able to sign an informed consent prior to treatment.
- A documented diagnosis of Fabry disease.
- Preferably two, but at minimum 1, historical serum creatinine evaluations in the last 2 years with the latest value within the last 6 months.
- Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms. Contraception should be used for 90 days after treatment discontinuation.
You may not qualify if:
- Patients enrolled and currently treated in Study PB-102-F20, and patients enrolled and currently treated in Extension Study PB-102-F60
- Patients who currently are on treatment under any other ongoing clinical trials of PRX-102
- History of Type 1 (anaphylaxis or anaphylactoid like) life-threatening hypersensitivity during previous exposure to other ERTs which could not be handled with medication
- Women who are breastfeeding may not participate unless they agree to stop breastfeeding.
- Women who are currently pregnant.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Chiesi Farmaceutici S.p.A.lead
- Chiesi USA, Inc.collaborator
Study Sites (11)
University of Alabama-Birmingham
Birmingham, Alabama, 35294, United States
Phoenix Children's Hospital, Inc.
Phoenix, Arizona, 85006, United States
University of California Irvine
Orange, California, 92868, United States
Central Coas Nephrology
Salinas, California, 93901, United States
University of Florida, Division of Pediatric Genetics
Jacksonville, Florida, 32207, United States
Emory University School of Medicine
Atlanta, Georgia, 30322, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Infusion Associates
Grand Rapids, Michigan, 49525, United States
Dallas Nephrology Associates
Dallas, Texas, 75235-2208, United States
University of Utah
Salt Lake City, Utah, 84108, United States
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
Fairfax, Virginia, 22030, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- expanded access
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 11, 2020
First Posted
September 17, 2020
Last Updated
July 31, 2024
Record last verified: 2024-07