Clinical trial NCT04552691: Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients
Official title: Expanded Access Treatment With Open-Label Pegunigalsidase Alfa for Fabry Patients
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Fabry Disease
Keywords: Fabry Disease
Pegunigalsidase alfa
Lead sponsor: Chiesi Farmaceutici S.p.A.
Sponsor class: INDUSTRY
Collaborators: name: Chiesi USA, Inc.
First posted: 2020-09-17
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT

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.


Eligibility 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.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama-Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital, Inc.
state: Arizona
city: Orange
country: United States
facility: University of California Irvine
state: California
city: Salinas
country: United States
facility: Central Coas Nephrology
state: California
city: Jacksonville
country: United States
facility: University of Florida, Division of Pediatric Genetics
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Dallas
country: United States
facility: Dallas Nephrology Associates
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Fairfax
country: United States
facility: Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
state: Virginia
Study officials:
References: