- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04552691
Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
March 28, 2023 updated by: Chiesi Farmaceutici S.p.A.
Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
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.
Study Overview
Study Type
Expanded Access
Expanded Access Type
- Intermediate-size Population
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Contact
- Name: Medical Information Chiesi
- Phone Number: (888) 661-9260
- Email: us.medical@chiesi.com
Study Locations
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Alabama
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Birmingham, Alabama, United States, 35294
- Available
- University of Alabama-Birmingham
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Contact:
- Eric Wallace
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Arizona
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Phoenix, Arizona, United States, 85006
- Available
- Phoenix Children's Hospital, Inc.
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Contact:
- Jasmine Knoll
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California
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Orange, California, United States, 92868
- Available
- University of California Irvine
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Contact:
- Virginia Kimonis
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Salinas, California, United States, 93901
- Available
- Central Coas Nephrology
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Contact:
- Barbara Rever
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Florida
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Jacksonville, Florida, United States, 32207
- Available
- University of Florida, Division of Pediatric Genetics
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Contact:
- Estella Mellin
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Georgia
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Atlanta, Georgia, United States, 30322
- Available
- Emory University School of Medicine
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Contact:
- William Wilcox
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Iowa
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Iowa City, Iowa, United States, 52242
- Available
- University of Iowa
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Contact:
- John Bernat
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Michigan
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Grand Rapids, Michigan, United States, 49525
- Available
- Infusion Associates
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Contact:
- Khan Nedd
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Texas
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Dallas, Texas, United States, 75235-2208
- Available
- Dallas Nephrology Associates
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Contact:
- Ankit Mehta
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Utah
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Salt Lake City, Utah, United States, 84108
- Available
- University of Utah
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Contact:
- Nicola Longo
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Virginia
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Fairfax, Virginia, United States, 22030
- Available
- Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
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Contact:
- Ozlem Goker-Alpan
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
December 7, 2022
Primary Completion
December 7, 2022
Study Completion
December 7, 2022
Study Registration Dates
First Submitted
September 11, 2020
First Submitted That Met QC Criteria
September 11, 2020
First Posted (Actual)
September 17, 2020
Study Record Updates
Last Update Posted (Actual)
March 31, 2023
Last Update Submitted That Met QC Criteria
March 28, 2023
Last Verified
March 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Metabolic Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Cerebral Small Vessel Diseases
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Fabry Disease
Other Study ID Numbers
- PB-102-F90
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
product manufactured in and exported from the U.S.
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Fabry Disease
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CENTOGENE GmbH RostockCompletedFabry Disease | Anderson-Fabry Disease | Fabry´s DiseaseArgentina, Belgium, Croatia, Czechia, Denmark, France, Germany, United Kingdom
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Wuerzburg University HospitalTakedaEnrolling by invitationLysosomal Storage Diseases | Fabry Disease | Fabry Disease, Cardiac Variant | HCM - Hypertrophic Cardiomyopathy | Anderson Fabry DiseaseGermany
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University Hospital, RouenUnknownAnderson-Fabry DiseaseFrance
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Sangamo TherapeuticsEnrolling by invitationFabry Disease | Fabry Disease, Cardiac VariantUnited States, Australia, United Kingdom
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Amicus Therapeutics France SASActive, not recruitingFabry Disease | Anderson Fabry DiseaseFrance
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Academisch Medisch Centrum - Universiteit van Amsterdam...RecruitingFabry Disease | Fabry Disease, Cardiac VariantNetherlands
-
Taipei Veterans General Hospital, TaiwanSanofiUnknownFabry Disease, Cardiac Variant
-
University of CambridgeSanofiRecruiting
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Shaare Zedek Medical CenterJohannes Gutenberg University MainzCompleted
-
Wolfson Medical CenterUnknownFabry Disease in the Young StrokeIsrael
Clinical Trials on Pegunigalsidase Alfa
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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Westfälische Wilhelms-Universität MünsterChiesi GmbHRecruiting
-
ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Spain, United Kingdom, Paraguay
-
Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseUnited States, Netherlands, Australia, Hungary, United Kingdom, Canada, Czechia, Finland, France, Italy, Norway, Slovenia, Spain
-
ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
-
Chiesi Farmaceutici S.p.A.Active, not recruitingFabry DiseaseBelgium, United States, United Kingdom, Italy, Czechia, Denmark, Norway
-
ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
-
ProtalixWithdrawn
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Chiesi Farmaceutici S.p.A.ICON plcNot yet recruiting
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Chiesi Farmaceutici S.p.A.ICON plcRecruiting