Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alfa (PRX-102) in Patients with Fabry Disease

Phase 3

Recruiting

Conditions: Fabry disease
lysosomal storage disease
10027424

Registration Number: NL-OMON52441

Lead Sponsor: Chiesi Farmaceutici S.p.A

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Recruiting

Sex: Not specified

Target Recruitment: 5

Inclusion Criteria

1. Completion of study PB-102-F20 or PB-102-F30, or completed at least 48
months in study PB-102-F03.
2. The patient signs informed consent
3. Female patients and male patients whose co-partners are of childbearing
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.

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>SAFETY ENDPOINTS:<br /><br>Changes from baseline in:<br /><br>• Clinical laboratory tests<br /><br>• Physical examination<br /><br>• Assessment of the injection site<br /><br>• Electrocardiography (ECG)<br /><br>• Brain MRI<br /><br>• Treatment-emergent adverse events (TEAE)<br /><br>• Ability to taper off infusion pre-medication at the start of the study<br /><br>• Requirement for use of pre-medication overall to manage infusion reactions<br /><br>• Treatment-emergent anti-pegunigalsidase alfa antibodies</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>EFFICACY ENDPOINTS:<br /><br>• Estimated glomerular filtration rate (eGFRCKD-EPI)<br /><br>• Left Ventricular Mass Index (g/m2) by magnetic resonance imaging (MRI)<br /><br>• Plasma Lyso-Gb3<br /><br>• Plasma Gb3 concentration<br /><br>• Protein/Creatinine ratio, spot urine test (UPCR)<br /><br>• Frequency of pain medication use<br /><br>• Exercise tolerance (Stress Test)<br /><br>• Short Form Brief Pain Inventory (BPI)<br /><br>• Mainz Severity Score Index (MSSI)<br /><br>• Quality of life (EQ-5D-5L)<br /><br>• Fabry disease clinical events</p><br>