Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234 | Active Not Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: CLI-06657AA1-03
• Study Type: interventional
• Target: 29
• Locations: 7 countries
• Sites: 14

Brief Summary

The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.

Conditions

Fabry Disease

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Evaluation of treatment-related adverse events

  CTCAE v4.03

  Throughout the study, 364 weeks

Secondary Outcomes (8)

• Kidney function 1

  Every 6 months throughout the duration of the study, 364 weeks

• Cardiac assessment

  Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364

• Biomarkers for Fabry disease

  Every 6 months throughout the duration of the study, 364 weeks

• Kidney function 2

  Every 6 months throughout the duration of the study, 364 weeks

• Clinical assessment

  Every four weeks throughout the duration of the study, 364 weeks

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+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Completion of study PB-102-F50.
• The patient signs informed consent.
• Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. 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.

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 (14)

UAB Medicine

Birmingham, Alabama, 35233, United States

Location

Emory University School of Medicine

Atlanta, Georgia, 30307, United States

Location

University of Iowa Hospitals and Clinica

Iowa City, Iowa, 52242, United States

Location

Infusion Associates

Grand Rapids, Michigan, 49525, United States

Location

Renal Disease Research Institute, LLC

Dallas, Texas, 75235, United States

Location

University of Utah Hospitals & Clinics

Salt Lake City, Utah, 84112, United States

Location

O & O Alpan

Fairfax, Virginia, 22030, United States

Location

UZ Antwerpen

Edegem, 2650, Belgium

Location

Fakultní poliklinika Všeobecné fakultní nemocnice v Praze

Prague, 128 08, Czechia

Location

Medical Endocrinology PE 2132, Rigshospitalet

Copenhagen, 2100, Denmark

Location

Azienda Ospedaliera Universitaria "Federico II"

Naples, Via Pansini, 80131, Italy

Location

Helse Bergen HF Haukeland Universitetssykehus

Bergen, 5021, Norway

Location

Addenbrooke's Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

The Royal Free Hospital

London, 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

Study Design

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

Model Details: Open-label extension study

Study Record Dates

• First Submitted: July 30, 2018
• First Posted: August 3, 2018
• Study Start: November 13, 2018
• Primary Completion: April 30, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: January 21, 2026

Record last verified: 2026-01

Locations

CZ (1); US (7); GB (2); IT (1); DK (1); BE (1); NO (1)