Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

NCT ID: NCT03566017

Last Updated: 2025-05-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

97 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-09-16

Study Completion Date

2025-01-21

Brief Summary

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The objective of CLI-06657AA1-04 (formerly PB-102-F60) is to evaluate the long-term safety, tolerability, and efficacy parameters of 1 mg/kg pegunigalsidase alfa administered intravenously every other week in adult Fabry patients who have successfully completed studies PB-102-F03, PB-102-F20 or PB-102-F30.

Detailed Description

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This is an open-label study. Patients will be enrolled to receive 1 mg/kg pegunigalsidase alfa as intravenous infusions every 2 weeks (±3 days). The duration of treatment is until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor. For the analysis, available efficacy and safety parameters will be summarized using descriptive statistics.

Conditions

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Fabry Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Open-label extension study
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Experimental open label

pegunigalsidase alfa

Group Type EXPERIMENTAL

pegunigalsidase alfa

Intervention Type DRUG

Recombinant human alpha galactosidase A

Interventions

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pegunigalsidase alfa

Recombinant human alpha galactosidase A

Intervention Type DRUG

Other Intervention Names

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PRX-102

Eligibility Criteria

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Inclusion Criteria

1. Completion of study PB-102-F20, PB-102-F03, or PB-102-F30
2. The patient signs informed consent
3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.

Exclusion Criteria

Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.
Minimum Eligible Age

18 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Chiesi Farmaceutici S.p.A.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UAB Medicine

Birmingham, Alabama, United States

Site Status

Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

University of California San Diego

La Jolla, California, United States

Site Status

University of California Irvine Center

Orange, California, United States

Site Status

University of Florida

Gainesville, Florida, United States

Site Status

Emory University School of Medicine

Atlanta, Georgia, United States

Site Status

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

Infusion Associates

Grand Rapids, Michigan, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

University of Pittsburgh

Pittsburgh, Pennsylvania, United States

Site Status

Renal Disease Research Institute, LLC - Dallas

Dallas, Texas, United States

Site Status

Institute of Metabolic Disease

Dallas, Texas, United States

Site Status

University of Utah Hospitals & Clinics

Salt Lake City, Utah, United States

Site Status

O+O Alpan LLC

Fairfax, Virginia, United States

Site Status

Medical College of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Royal Melbourne Hospital

Parkville, Victoria, Australia

Site Status

Capital District Health Authority

Halifax, Nova Scotia, Canada

Site Status

Vseobecna fakultni nemocnice v Praze

Prague, Czech Republic, Czechia

Site Status

Turku University Central Hospital

Turku, , Finland

Site Status

Hospital Raymond-Poincaré

Garches, , France

Site Status

Semmelweis Egyetem

Budapest, , Hungary

Site Status

Azienda Ospedaliera Universitaria "Federico II"

Napoli, Via Pansini, Italy

Site Status

Academisch Medisch Centrum

Amsterdam, , Netherlands

Site Status

Haukeland University Hospital Klinisk Forskningspost

Bergen, , Norway

Site Status

General Hospital Slovenj Gradec

Slovenj Gradec, , Slovenia

Site Status

Hospital de Dia Quiron Zaragoza

Zaragoza, , Spain

Site Status

University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

Edgbaston, Birmingham, United Kingdom

Site Status

Salford Royal

Salford, Greater Manchester, United Kingdom

Site Status

Addenbrooke's Hospital

Cambridge, , United Kingdom

Site Status

The Royal Free Hospital

London, , United Kingdom

Site Status

Countries

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United States Australia Canada Czechia Finland France Hungary Italy Netherlands Norway Slovenia Spain United Kingdom

Other Identifiers

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CLI-06657AA1-04

Identifier Type: -

Identifier Source: org_study_id

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