Safety and Efficacy study assessing Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Patients With Fabry Disease

Phase 1

• Conditions: MedDRA version: 24.1Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Body processes [G] - Genetic Phenomena [G05]

• Registration Number: EUCTR2018-001947-30-DK
• Lead Sponsor: Chiesi Farmaceutici S.p.A

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2019-06-13
• Last Update Posted: 15 July 2024

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 28

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 ccepted, highly effective 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 28
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified