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

Fabry Disease Clinical Trial

Official title:

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03566017
• Other study ID #: CLI-06657AA1-04
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: September 16, 2018
• Est. completion date: April 2025

Study information

• Verified date: April 2023
• Source: Chiesi Farmaceutici S.p.A.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 97
• Est. completion date: April 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• pegunigalsidase alfa
Recombinant human alpha galactosidase A

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital	Parkville	Victoria
Canada	Capital District Health Authority	Halifax	Nova Scotia
Czechia	Vseobecna fakultni nemocnice v Praze	Prague	Czech Republic
Finland	Turku University Central Hospital	Turku
France	Hospital Raymond-Poincaré	Garches
Hungary	Semmelweis Egyetem	Budapest
Italy	Azienda Ospedaliera Universitaria "Federico II"	Napoli	Via Pansini
Netherlands	Academisch Medisch Centrum	Amsterdam
Norway	Haukeland University Hospital Klinisk Forskningspost	Bergen
Slovenia	General Hospital Slovenj Gradec	Slovenj Gradec
Spain	Hospital de Dia Quiron Zaragoza	Zaragoza
United Kingdom	Addenbrooke's Hospital	Cambridge
United Kingdom	University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital	Edgbaston	Birmingham
United Kingdom	The Royal Free Hospital	London
United Kingdom	Salford Royal	Salford	Greater Manchester
United States	Emory University School of Medicine	Atlanta	Georgia
United States	UAB Medicine	Birmingham	Alabama
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Renal Disease Research Institute, LLC - Dallas	Dallas	Texas
United States	O+O Alpan LLC	Fairfax	Virginia
United States	University of Florida	Gainesville	Florida
United States	Infusion Associates	Grand Rapids	Michigan
United States	University of Iowa Hospitals and Clinics	Iowa City	Iowa
United States	University of California San Diego	La Jolla	California
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	University of California Irvine Center	Orange	California
United States	Phoenix Children's Hospital	Phoenix	Arizona
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	Eccles Primary Children's Outpatient Services Building	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of treatment-related adverse events	CTCAE v4.03	Throughout the study, 364 weeks
Secondary	Kidney function 1	Estimated glomerular filtration rate (eGFRCKD-EPI)	Every 6 months throughout trial, 364 weeks
Secondary	Cardiac assessment	Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI) and Echocardiograph, and exercise tolerance (Stress Test)	Every 12 months to end of the study, 7 years
Secondary	Biomarkers for Fabry disease	plasma Lyso-Gb3 and Gb3	Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
Secondary	Record of pain medication use	Frequency of pain medication use, or pre-infusion medication	Every two weeks for 7 years
Secondary	Kidney function 2	Protein/Creatinine ratio, spot urine test (UPCR)	Every 6 months to the end of the study, 7 years
Secondary	Pain assessment	short form Brief Pain Inventory (BPI)	Every 6 months up to the end of the study, 7 years
Secondary	Symptom assessment	Mainz Severity Score Index (MSSI)	Every 12 months up to the end of the study, 7 years
Secondary	Quality of life assessment	quality of life (EQ-5D-5L)	Every 6 months up to the end of the study, 7 years