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Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04552691
Expanded Access Status : Available
First Posted : September 17, 2020
Last Update Posted : March 31, 2023
Sponsor:
Collaborator:
Chiesi USA, Inc.
Information provided by (Responsible Party):
Chiesi Farmaceutici S.p.A.

Study Description
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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
Fabry Disease Drug: Pegunigalsidase Alfa

Study Design
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Study Type : Expanded Access
Expanded Access Type : Intermediate-size Population
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients

Resource links provided by the National Library of Medicine


Interventions
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Intervention Details:
  • Drug: Pegunigalsidase Alfa
    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
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Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04552691


Contacts
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Contact: Medical Information Chiesi (888) 661-9260 us.medical@chiesi.com

Locations
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United States, Alabama
University of Alabama-Birmingham Available
Birmingham, Alabama, United States, 35294
Contact: Eric Wallace         
United States, Arizona
Phoenix Children's Hospital, Inc. Available
Phoenix, Arizona, United States, 85006
Contact: Jasmine Knoll         
United States, California
University of California Irvine Available
Orange, California, United States, 92868
Contact: Virginia Kimonis         
Central Coas Nephrology Available
Salinas, California, United States, 93901
Contact: Barbara Rever         
United States, Florida
University of Florida, Division of Pediatric Genetics Available
Jacksonville, Florida, United States, 32207
Contact: Estella Mellin         
United States, Georgia
Emory University School of Medicine Available
Atlanta, Georgia, United States, 30322
Contact: William Wilcox         
United States, Iowa
University of Iowa Available
Iowa City, Iowa, United States, 52242
Contact: John Bernat         
United States, Michigan
Infusion Associates Available
Grand Rapids, Michigan, United States, 49525
Contact: Khan Nedd         
United States, Texas
Dallas Nephrology Associates Available
Dallas, Texas, United States, 75235-2208
Contact: Ankit Mehta         
United States, Utah
University of Utah Available
Salt Lake City, Utah, United States, 84108
Contact: Nicola Longo         
United States, Virginia
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC) Available
Fairfax, Virginia, United States, 22030
Contact: Ozlem Goker-Alpan         
Sponsors and Collaborators
Chiesi Farmaceutici S.p.A.
Chiesi USA, Inc.
More Information
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Responsible Party: Chiesi Farmaceutici S.p.A.
ClinicalTrials.gov Identifier: NCT04552691    
Other Study ID Numbers: PB-102-F90
First Posted: September 17, 2020    Key Record Dates
Last Update Posted: March 31, 2023
Last Verified: March 2023
Keywords provided by Chiesi Farmaceutici S.p.A.:
Fabry Disease
Pegunigalsidase alfa
Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
 Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders