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

NCT03566017 | Completed Phase 3 Results FDA Drug

• 1 other identifier: CLI-06657AA1-04
• Study Type: interventional
• Target: 97
• Locations: 13 countries
• Sites: 30

Brief Summary

The objective of CLI-06657AA1-04 (formerly PB-102-F60) was 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 had successfully completed studies PB-102-F20, PB-102-F30, or at least 48 months in study PB-102-F03.

Conditions

Fabry Disease

Keywords

Glomerular filtration rate; Proteinuria; PRX-102; pegunigalsidase alfa; Fabry disease

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Treatment-related Adverse Events

  No primary or secondary endpoints were specified for this trial. Evaluation of safety was a main objective. A treatment-emergent adverse event (TEAE) was defined as any adverse event (AE) occurring after the start of study treatment and within the time of residual drug effect (20 days after last administration of study treatment) or a pre-treatment AE or medical condition that worsened in severity after the start of study treatment and within the time of residual drug effect. Each TEAE was classified for severity based on the Common Terminology Criteria for Adverse Events (CTCAE) v4.03 and for causality in the categories unrelated, unlikely, possible, probable and definitely. Treatment-related AEs were TEAEs with causality assessed as possible, probable or definitely related to study treatment. TEAEs were coded using the Medical Dictionary for Regulatory Activities (MedDRA) version (v)19.0. Related TEAEs reported in ≥2 percent of participants by Preferred Term are reported.

  From first ever infusion of pegunigalsidase alfa, which could have been in the parent study (PB-102-F01/F20/F30) or this extension study, CLI-06657AA1-04, until 90 days after the final dose visit for each participant. Mean individual exposure: 5.5 years

Secondary Outcomes (17)

• Change From Baseline to the Last Observation for the eGFR

  From baseline (assessment before first exposure to study drug, which could have been in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)

• Annualized eGFR Slope

  From first ever infusion of pegunigalsidase alfa (which could have been in the parent study, PB-102-F01/F20/F30, or this extension study, CLI-06657AA1-04), until the end of the extension study; mean individual exposure: 5.5 years

• Shift From Baseline to the Last Scheduled Visit in UPCR Category

  Shift from baseline (assessment before first exposure to study drug which could be in the parent study PB-102-F01/F02/F30 or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)

• Change From Baseline to the Last Observation in Plasma Lyso-Gb3 and Globotriaosylceramide (Gb3) Concentrations

  Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04 ) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)

• Change From Baseline to the Last Observation for Pain Severity Items of the Brief Pain Inventory (BPI)

  Change from baseline (assessment before first exposure to study drug, which could be in the parent study, PB-102-F01/F20/F30, or CLI-06657AA1-04) to last observation (last non-missing assessment; Mean(SD) exposure: 5.5 (1.96) years; range:1.3;10.6 years)

Study Arms (1)

Experimental open label

EXPERIMENTAL

pegunigalsidase alfa

Drug: pegunigalsidase alfa

Interventions

pegunigalsidase alfa DRUG

Recombinant human alpha galactosidase A

Also known as: PRX-102

Experimental open label

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Completion of study PB-102-F20, study PB-102-F30, or at least 48 months in study PB-102-F03.
• The participant signed informed consent.
• Female participants and male participants whose co-partners were of child-bearing potential agreed to use a medically acceptable method of contraception. These included 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, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or sexual abstinence. Contraception had to be used for 2 weeks after treatment termination.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chiesi Farmaceutici S.p.A.: lead

Study Sites (30)

UAB Medicine

Birmingham, Alabama, 35233, United States

Location

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

University of California San Diego

La Jolla, California, 92037, United States

Location

University of California Irvine Center

Orange, California, 92868, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30322, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Infusion Associates

Grand Rapids, Michigan, 49525, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Renal Disease Research Institute, LLC - Dallas

Dallas, Texas, 75235, United States

Location

Institute of Metabolic Disease

Dallas, Texas, 75246, United States

Location

University of Utah Hospitals & Clinics

Salt Lake City, Utah, 84108, United States

Location

O+O Alpan LLC

Fairfax, Virginia, 22030, United States

Location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

Location

Royal Melbourne Hospital

Parkville, Victoria, 3050, Australia

Location

Capital District Health Authority

Halifax, Nova Scotia, B3H 1V8, Canada

Location

Vseobecna fakultni nemocnice v Praze

Prague, Czech Republic, 12808, Czechia

Location

Turku University Central Hospital

Turku, FI-20521, Finland

Location

Hospital Raymond-Poincaré

Garches, 92380, France

Location

Semmelweis Egyetem

Budapest, 1083, Hungary

Location

Azienda Ospedaliera Universitaria "Federico II"

Naples, Via Pansini, 80131, Italy

Location

Academisch Medisch Centrum

Amsterdam, 1105 AZ, Netherlands

Location

Haukeland University Hospital Klinisk Forskningspost

Bergen, 5021, Norway

Location

General Hospital Slovenj Gradec

Slovenj Gradec, 2380, Slovenia

Location

Hospital de Dia Quiron Zaragoza

Zaragoza, 50012, Spain

Location

University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital

Edgbaston, Birmingham, B152TH, United Kingdom

Location

Salford Royal

Salford, Greater Manchester, M6 8HD, United Kingdom

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

The Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

MeSH Terms

Conditions

Fabry Disease; Proteinuria

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders; Urination Disorders; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Clinical Trial Transparency
• Organization: Chiesi Farmaceutici S.p.A.

clinicaltrials_info@chiesi.com

+3905212791

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Model Details: Open-label extension study

Study Record Dates

• First Submitted: May 24, 2018
• First Posted: June 21, 2018
• Study Start: September 16, 2018
• Primary Completion: January 21, 2025
• Study Completion: January 21, 2025
• Last Updated: March 24, 2026
• Results First Posted: March 24, 2026

Record last verified: 2026-03

Locations

NL (1); HU (1); US (15); CZ (1); SL (1); ES (1); GB (4); FR (1); CA (1); IT (1); NO (1); FI (1); AU (1)