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

Phase 3

Active, not recruiting

• Conditions: Fabry Disease
• Interventions: Drug: pegunigalsidase alfa

• Registration Number: NCT03614234
• Lead Sponsor: Chiesi Farmaceutici S.p.A.

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.

Detailed Description

This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.

Study Timeline

• Study First Submitted: 2018-07-30
• Study First Submitted QC: 2018-08-02
• Study First Posted: 2018-08-03
• Study Start: 2018-11-13
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-07
• Last Update Posted: 2024-10-08
• Primary Completion: 2025-12-31
• Study Completion: 2026-03-28

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 29

Inclusion Criteria

1. Completion of study PB-102-F50.
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 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.

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Experimental open label	pegunigalsidase alfa	Pegunigalsidase alfa

Primary Outcome Measures

Name	Time	Method
Evaluation of treatment-related adverse events	Throughout the study, 364 weeks	CTCAE v4.03

Secondary Outcome Measures

Name	Time	Method
Biomarkers for Fabry disease	Every 6 months throughout the duration of the study, 364 weeks	Plasma Lyso-Gb3 and Gb3
Pain assessment	Every 6 months throughout the duration of the study, 364 weeks	Short form Brief Pain Inventory (BPI)
Cardiac assessment	Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364	Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Kidney function 1	Every 6 months throughout the duration of the study, 364 weeks	Estimated glomerular filtration rate (eGFRCKD-EPI)
Kidney function 2	Every 6 months throughout the duration of the study, 364 weeks	Protein/Creatinine ratio (UPCR), spot urine test
Clinical assessment	Every four weeks throughout the duration of the study, 364 weeks	Record of pain medication and pre-medication use
Symptom assessment	Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364	Mainz Severity Score Index (MSSI)
Quality of life assessment	Every 6 months throughout the duration of the study, 364 weeks	Quality of life (EQ-5D-5L)

Trial Locations

Locations (14)

Renal Disease Research Institute, LLC; 🇺🇸 Dallas, Texas, United States

Medical Endocrinology PE 2132, Rigshospitalet; 🇩🇰 Copenhagen, Denmark

UAB Medicine; 🇺🇸 Birmingham, Alabama, United States

University of Iowa Hospitals and Clinica; 🇺🇸 Iowa City, Iowa, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

University of Utah Hospitals & Clinics; 🇺🇸 Salt Lake City, Utah, United States

O & O Alpan; 🇺🇸 Fairfax, Virginia, United States

UZ Antwerpen; 🇧🇪 Edegem, Belgium

Helse Bergen HF Haukeland Universitetssykehus; 🇳🇴 Bergen, Norway

Azienda Ospedaliera Universitaria "Federico II"; 🇮🇹 Napoli, Via Pansini, Italy

Fakultní poliklinika Všeobecné fakultní nemocnice v Praze; 🇨🇿 Praha, Czechia

The Royal Free Hospital; 🇬🇧 London, United Kingdom

Infusion Associates; 🇺🇸 Grand Rapids, Michigan, United States

Addenbrooke's Hospital; 🇬🇧 Cambridge, United Kingdom