Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 1 May 2023
• Main ID: NCT03566017
• Date of registration: 24/05/2018
• Prospective Registration: Yes
• Primary sponsor: Chiesi Farmaceutici S.p.A.
• Public title: Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease
• Scientific title: Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients With Fabry Disease
• Date of first enrolment: September 16, 2018
• Target sample size: 97
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03566017
• 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

Australia, Canada, Czechia, Finland, France, Hungary, Italy, Netherlands, Norway, Slovenia, Spain, United Kingdom, United States

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 60 Years
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)

Kidney function 1 [Time Frame: Every 6 months throughout trial, 364 weeks]

Record of pain medication use [Time Frame: Every two weeks for 7 years]

Quality of life assessment [Time Frame: Every 6 months up to the end of the study, 7 years]

Symptom assessment [Time Frame: Every 12 months up to the end of the study, 7 years]

Biomarkers for Fabry disease [Time Frame: Every 12 months to end of the study, 7 years. For patients from PB-102-F20 at 3 and 6 months.]

Cardiac assessment [Time Frame: Every 12 months to end of the study, 7 years]

Pain assessment [Time Frame: Every 6 months up to the end of the study, 7 years]

Kidney function 2 [Time Frame: Every 6 months to the end of the study, 7 years]

Secondary ID(s)

CLI-06657AA1-04

Source(s) of Monetary Support

Please refer to primary and secondary sponsors