Clinical trial NCT03614234: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
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: 2018-08-03
Enrollment: 29
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_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-06657AA1-03 (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.

Eligibility criteria: 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.

Primary outcomes:
measure: Evaluation of treatment-related adverse events
timeFrame: Throughout the study, 364 weeks
Secondary outcomes:
measure: Kidney function 1
timeFrame: Every 6 months throughout the duration of the study, 364 weeks
measure: Cardiac assessment
timeFrame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
measure: Biomarkers for Fabry disease
timeFrame: Every 6 months throughout the duration of the study, 364 weeks
measure: Kidney function 2
timeFrame: Every 6 months throughout the duration of the study, 364 weeks
measure: Clinical assessment
timeFrame: Every four weeks throughout the duration of the study, 364 weeks
measure: Pain assessment
timeFrame: Every 6 months throughout the duration of the study, 364 weeks
measure: Symptom assessment
timeFrame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364
measure: Quality of life assessment
timeFrame: Every 6 months throughout the duration of the study, 364 weeks
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: UAB Medicine
state: Alabama
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 Clinica
state: Iowa
city: Grand Rapids
country: United States
facility: Infusion Associates
state: Michigan
city: Dallas
country: United States
facility: Renal Disease Research Institute, LLC
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
state: Virginia
city: Edegem
country: Belgium
facility: UZ Antwerpen
city: Praha
country: Czechia
facility: Fakultní poliklinika Všeobecné fakultní nemocnice v Praze
city: Copenhagen
country: Denmark
facility: Medical Endocrinology PE 2132, Rigshospitalet
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II"
state: Via Pansini
city: Bergen
country: Norway
facility: Helse Bergen HF Haukeland Universitetssykehus
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
city: London
country: United Kingdom
facility: The Royal Free Hospital
Study officials:
References: