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

Fabry Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Available

Administrative data

• NCT number: NCT04552691
• Other study ID #: PB-102-F90
• Status: Available

Study information

• Verified date: March 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: Medical Information Chiesi
• Phone: (888) 661-9260
• Email: us.medical[@]chiesi.com
• Is FDA regulated: No
• Study type: Expanded Access

Clinical Trial 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.

Recruitment information / eligibility

• Status: Available
• Enrollment: 0
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

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).

Locations

Country	Name	City	State
United States	Emory University School of Medicine	Atlanta	Georgia
United States	University of Alabama-Birmingham	Birmingham	Alabama
United States	Dallas Nephrology Associates	Dallas	Texas
United States	Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)	Fairfax	Virginia
United States	Infusion Associates	Grand Rapids	Michigan
United States	University of Iowa	Iowa City	Iowa
United States	University of Florida, Division of Pediatric Genetics	Jacksonville	Florida
United States	University of California Irvine	Orange	California
United States	Phoenix Children's Hospital, Inc.	Phoenix	Arizona
United States	Central Coas Nephrology	Salinas	California
United States	University of Utah	Salt Lake City	Utah

Sponsors (2)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.	Chiesi USA, Inc.

Country where clinical trial is conducted

United States,