Bright51: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Sponsor

Protalix (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT03614234

Collaborator

(none)

Enrollment

Locations

Arm

70.8

Anticipated Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The objective of 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.

Condition or Disease	Intervention/Treatment	Phase
Fabry Disease	Biological: pegunigalsidase alfa	Phase 3

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 up to 48 months or until pegunigalsidase alfa is available to the patient at the discretion of the Sponsor. Interim analyses may be performed for administrative purposes during the conduct of the study.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

Open-label extension studyOpen-label extension study

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Nov 6, 2018

Anticipated Primary Completion Date :

Aug 1, 2024

Anticipated Study Completion Date :

Oct 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Experimental open label Pegunigalsidase alfa	Biological: pegunigalsidase alfa Recombinant human alpha galactosidase A Other Names: PRX-102

Arm

Intervention/Treatment

Experimental: Experimental open label

Pegunigalsidase alfa

Biological: pegunigalsidase alfa

Recombinant human alpha galactosidase A

Other Names:

PRX-102

Outcome Measures

Primary Outcome Measures

Evaluation of treatment-related adverse events [Throughout the study, 208 weeks]
CTCAE v4.03

Secondary Outcome Measures

Kidney function [At 56 and108 weeks and at the end of the study, 208 weeks]
Estimated glomerular filtration rate (eGFRCKD-EPI)
Cardiac assessment [At 56 and 108 weeks and at the end of the study, 208 weeks]
Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test
Biomarkers for Fabry disease [At 56 and 108 weeks and at the end of the study, 208 weeks]
Plasma Lyso-Gb3
Clinical assessment [Every four weeks throughout the duration of the study, 208 weeks]
Record of pain medication use
Kidney function [At 56 and 108 weeks and at the end of the study, 208 weeks]
Protein/Creatinine ratio (UPCR), spot urine test
Pain assessment [At 56 and 108 weeks and at the end of the study, 208 weeks]
Short form Brief Pain Inventory (BPI)
Symptom assessment [At 56 and 108 weeks and at the end of the study, 208 weeks]
Mainz Severity Score Index (MSSI)
Quality of life assessment [At 56 and 108 weeks and at the end of the study, 208 weeks]
Quality of life (EQ-5D-5L)

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

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, highly effective method of contraception. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence.

Exclusion Criteria:

Presence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with patient compliance with the requirements of the study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UAB Medicine	Birmingham	Alabama	United States	35233
2	Emory University School of Medicine	Atlanta	Georgia	United States	30307
3	University of Iowa Hospitals and Clinica	Iowa City	Iowa	United States	52242
4	Infusion Associates	Grand Rapids	Michigan	United States	49525
5	Renal Disease Research Institute, LLC	Dallas	Texas	United States	75235
6	University of Utah Hospitals & Clinics	Salt Lake City	Utah	United States	84112
7	O & O Alpan	Fairfax	Virginia	United States	22030
8	UZ Antwerpen	Edegem		Belgium	2650
9	Fakultní poliklinika Všeobecné fakultní nemocnice v Praze	Praha		Czechia	128 08
10	Medical Endocrinology PE 2132, Rigshospitalet	Copenhagen		Denmark	2100
11	Azienda Ospedaliera Universitaria "Federico II"	Napoli		Italy	80131
12	Helse Bergen HF Haukeland Universitetssykehus	Bergen		Norway	5021
13	Hospital Quirón Floresta	Zaragoza		Spain	50012
14	Addenbrooke's Hospital	Cambridge		United Kingdom	CB2 0QQ
15	The Royal Free Hospital	London		United Kingdom