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

• Register: ClinicalTrials.gov
• Last refreshed on: 1 May 2023
• Main ID: NCT03614234
• Date of registration: 30/07/2018
• Prospective Registration: Yes
• Primary sponsor: Chiesi Farmaceutici S.p.A.
• Public title: Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
• Scientific 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
• Date of first enrolment: November 13, 2018
• Target sample size: 29
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03614234
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Belgium, Czechia, Denmark, Italy, Norway, Spain, United Kingdom, United States

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: pegunigalsidase alfa

Primary Outcome(s)

Evaluation of treatment-related adverse events [Time Frame: Throughout the study, 364 weeks]

Secondary Outcome(s)

Biomarkers for Fabry disease [Time Frame: Every 6 months throughout the duration of the study, 364 weeks]

Symptom assessment [Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364]

Cardiac assessment [Time Frame: Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364]

Kidney function 1 [Time Frame: Every 6 months throughout the duration of the study, 364 weeks]

Kidney function 2 [Time Frame: Every 6 months throughout the duration of the study, 364 weeks]

Pain assessment [Time Frame: Every 6 months throughout the duration of the study, 364 weeks]

Clinical assessment [Time Frame: Every four weeks throughout the duration of the study, 364 weeks]

Quality of life assessment [Time Frame: Every 6 months throughout the duration of the study, 364 weeks]

Secondary ID(s)

CLI-06657AA1-03

Source(s) of Monetary Support

Please refer to primary and secondary sponsors