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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. Identifier: NCT04552691
Expanded Access Status : Available
First Posted : September 17, 2020
Last Update Posted : March 31, 2023
Chiesi USA, Inc.
Information provided by (Responsible Party):
Chiesi Farmaceutici S.p.A.

Tracking Information
First Submitted Date September 11, 2020
First Posted Date September 17, 2020
Last Update Posted Date March 31, 2023
Descriptive Information
Brief Title Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
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.
Detailed Description Not Provided
Study Type Expanded Access
Expanded Access Type Intermediate-size Population
Intervention 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).
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Expanded Access Status Available
Contact: Medical Information Chiesi (888) 661-9260
Listed Location Countries United States
Removed Location Countries  
Administrative Information
NCT Number NCT04552691
Current Responsible Party Chiesi Farmaceutici S.p.A.
Original Responsible Party Protalix
Current Study Sponsor Chiesi Farmaceutici S.p.A.
Original Study Sponsor Protalix
Collaborators Chiesi USA, Inc.
Investigators Not Provided
PRS Account Chiesi Farmaceutici S.p.A.
Verification Date March 2023