Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Study Details
Study Description
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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Study Design
Outcome Measures
Primary Outcome Measures
Eligibility Criteria
Criteria
Inclusion Criteria:
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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.
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Patient (or legal guardian) is able to sign an informed consent prior to treatment.
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A documented diagnosis of Fabry disease.
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Preferably two, but at minimum 1, historical serum creatinine evaluations in the last 2 years with the latest value within the last 6 months.
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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.
Exclusion Criteria:
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Patients enrolled and currently treated in Study PB-102-F20, and patients enrolled and currently treated in Extension Study PB-102-F60
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Patients who currently are on treatment under any other ongoing clinical trials of PRX-102
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History of Type 1 (anaphylaxis or anaphylactoid like) life-threatening hypersensitivity during previous exposure to other ERTs which could not be handled with medication
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Women who are breastfeeding may not participate unless they agree to stop breastfeeding.
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Women who are currently pregnant.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of Alabama-Birmingham | Birmingham | Alabama | United States | 35294 |
| 2 | Phoenix Children's Hospital, Inc. | Phoenix | Arizona | United States | 85006 |
| 3 | University of Florida, Division of Pediatric Genetics | Jacksonville | Florida | United States | 32207 |
| 4 | Emory University School of Medicine | Atlanta | Georgia | United States | 30322 |
| 5 | University of Iowa | Iowa City | Iowa | United States | 52242 |
| 6 | Infusion Associates | Grand Rapids | Michigan | United States | 49525 |
| 7 | The Stern Cardiovascular Foundation, Inc. | Memphis | Tennessee | United States | 38163 |
| 8 | Dallas Nephrology Associates | Dallas | Texas | United States | 75235-2208 |
| 9 | University of Utah | Salt Lake City | Utah | United States | 84108 |
| 10 | Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC) | Fairfax | Virginia | United States | 22030 |
| 11 | West Virginia University Research Corporation | Morgantown | West Virginia | United States | 26506 |
Sponsors and Collaborators
- Protalix
- Chiesi USA, Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- PB-102-F90