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

Primary Purpose

Fabry Disease

Status
Available
Phase
Locations
United States
Study Type
Expanded Access
Intervention
Pegunigalsidase Alfa
Sponsored by
Chiesi Farmaceutici S.p.A.
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Fabry Disease focused on measuring Fabry Disease, Pegunigalsidase alfa

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

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.

Sites / Locations

  • University of Alabama-Birmingham
  • Phoenix Children's Hospital, Inc.
  • University of California Irvine
  • Central Coas Nephrology
  • University of Florida, Division of Pediatric Genetics
  • Emory University School of Medicine
  • University of Iowa
  • Infusion Associates
  • Dallas Nephrology Associates
  • University of Utah
  • Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
September 11, 2020
Last Updated
March 28, 2023
Sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
Chiesi USA, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04552691
Brief Title
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

2. Study Status

Record Verification Date
March 2023
Overall Recruitment Status
Available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chiesi Farmaceutici S.p.A.
Collaborators
Chiesi USA, Inc.

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
Fabry Disease, Pegunigalsidase alfa

7. Study Design

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Pegunigalsidase Alfa
Intervention Description
Pegunigalsidase alfa is a recombinant ERT (enzyme replacement therapy) used to treat Fabry disease (dosage: 1 mg/kg body weight every 2 weeks).

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Medical Information Chiesi
Phone
(888) 661-9260
Email
us.medical@chiesi.com
Facility Information:
Facility Name
University of Alabama-Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35294
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Eric Wallace
Facility Name
Phoenix Children's Hospital, Inc.
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jasmine Knoll
Facility Name
University of California Irvine
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Virginia Kimonis
Facility Name
Central Coas Nephrology
City
Salinas
State/Province
California
ZIP/Postal Code
93901
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Barbara Rever
Facility Name
University of Florida, Division of Pediatric Genetics
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32207
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Estella Mellin
Facility Name
Emory University School of Medicine
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
William Wilcox
Facility Name
University of Iowa
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
John Bernat
Facility Name
Infusion Associates
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49525
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Khan Nedd
Facility Name
Dallas Nephrology Associates
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235-2208
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ankit Mehta
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nicola Longo
Facility Name
Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC)
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Individual Site Status
Available
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ozlem Goker-Alpan

12. IPD Sharing Statement

Learn more about this trial

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

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