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

Fabry Disease Clinical Trial

Official title:

Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03614234
• Other study ID #: CLI-06657AA1-03
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 13, 2018
• Est. completion date: December 2024

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-06657AA1-03 (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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 29
• Est. completion date: December 2024
• Est. primary completion date: September 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Fabry Disease

Intervention

Drug:
• pegunigalsidase alfa
Recombinant human alpha galactosidase A

Locations

Country	Name	City	State
Belgium	UZ Antwerpen	Edegem
Czechia	Fakultní poliklinika Všeobecné fakultní nemocnice v Praze	Praha
Denmark	Medical Endocrinology PE 2132, Rigshospitalet	Copenhagen
Italy	Azienda Ospedaliera Universitaria "Federico II"	Napoli	Via Pansini
Norway	Helse Bergen HF Haukeland Universitetssykehus	Bergen
United Kingdom	Addenbrooke's Hospital	Cambridge
United Kingdom	The Royal Free Hospital	London
United States	Emory University School of Medicine	Atlanta	Georgia
United States	UAB Medicine	Birmingham	Alabama
United States	Renal Disease Research Institute, LLC	Dallas	Texas
United States	O & O Alpan	Fairfax	Virginia
United States	Infusion Associates	Grand Rapids	Michigan
United States	University of Iowa Hospitals and Clinica	Iowa City	Iowa
United States	University of Utah Hospitals & Clinics	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Chiesi Farmaceutici S.p.A.

Countries where clinical trial is conducted

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