Clinical trial NCT03566017: Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
Official title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Fabry Disease
Keywords: Glomerular filtration rate
Proteinuria
PRX-102
pegunigalsidase alfa
Fabry disease
Lead sponsor: Chiesi Farmaceutici S.p.A.
Sponsor class: INDUSTRY
First posted: 2018-06-21
Enrollment: 97
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Open-label extension study
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluation of treatment-related adverse events
timeFrame: Throughout the study, 7 years
Secondary outcomes:
measure: Kidney function 1
timeFrame: Every 6 months throughout trial, 7 years
measure: Cardiac assessment
timeFrame: Every 12 months to end of the study, 7 years
measure: Biomarkers for Fabry disease
timeFrame: Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.
measure: Record of pain medication use
timeFrame: Every two weeks for 7 years
measure: Kidney function 2
timeFrame: Every 6 months to the end of the study, 7 years
measure: Pain assessment
timeFrame: Every 6 months up to the end of the study, 7 years
measure: Symptom assessment
timeFrame: Every 12 months up to the end of the study, 7 years
measure: Quality of life assessment
timeFrame: Every 6 months up to the end of the study, 7 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: UAB Medicine
state: Alabama
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: Orange
country: United States
facility: University of California Irvine Center
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: Renal Disease Research Institute, LLC - Dallas
state: Texas
city: Dallas
country: United States
facility: Institute of Metabolic Disease
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah Hospitals & Clinics
state: Utah
city: Fairfax
country: United States
facility: O+O Alpan LLC
state: Virginia
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Parkville
country: Australia
facility: Royal Melbourne Hospital
state: Victoria
city: Halifax
country: Canada
facility: Capital District Health Authority
state: Nova Scotia
city: Prague
country: Czechia
facility: Vseobecna fakultni nemocnice v Praze
state: Czech Republic
city: Turku
country: Finland
facility: Turku University Central Hospital
city: Garches
country: France
facility: Hospital Raymond-Poincaré
city: Budapest
country: Hungary
facility: Semmelweis Egyetem
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II"
state: Via Pansini
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum
city: Bergen
country: Norway
facility: Haukeland University Hospital Klinisk Forskningspost
city: Slovenj Gradec
country: Slovenia
facility: General Hospital Slovenj Gradec
city: Zaragoza
country: Spain
facility: Hospital de Dia Quiron Zaragoza
city: Edgbaston
country: United Kingdom
facility: University Hospitals Birmingham NHS Foundation Trust, Queen Elizabeth Hospital
state: Birmingham
city: Salford
country: United Kingdom
facility: Salford Royal
state: Greater Manchester
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
city: London
country: United Kingdom
facility: The Royal Free Hospital
Study officials:
References: