MedDRA version: 24.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Chiesi Farmaceutici S.p.A.
Eligibility
Sex/Gender
All
Inclusion 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, 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 made condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably made 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
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the ongoing safety and efficacy parameters of 2 mg/kg pegunigalsidase alfa every 4 weeks in adult Fabry patients.;Secondary Objective: N/A;Primary end point(s): SAFETY ENDPOINTS: Changes from baseline in: *Clinical laboratory tests *Physical examination *Assessment of the injection site *Electrocardiogram *Brain MRI *Treatment-emergent adverse events *Requirement for use of pre-medication overall to manage infusion reactions *Treatment-induced anti-pegunigalsidase alfa antibodies;Timepoint(s) of evaluation of this end point: Results of safety evaluations (including TEAEs, injection site reactions, physical examinations, ECG, laboratory analyses, and anti-drug antibodies) will be summarized and described. No formal statistical analyses will be performed in this study. The study endpoints will be evaluated by various summary analyses by study visit and by change from baseline data for each efficacy endpoint. Safety and efficacy endpoints will be compared to baseline using summary statistics. Data will not be analysed by inferential statistics. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): EFFICACY EXPLORATORY ENDPOINTS: * Estimated glomerular filtration rate (eGFRCKD-EPI). * Left Ventricular Mass Index (g/m2) by echocardiogram. * Plasma Gb3 concentration. * Plasma Lyso-Gb3 concentration. * Protein/Creatinine ratio, spot urine test. * Frequency of pain medication use. * Exercise tolerance (Stress Test). * Short Form Brief Pain Inventory (BPI). * Mainz Severity Score Index (MSSI). * Quality of Life (EQ-5D-5L).;Timepoint(s) of evaluation of this end point: The study endpoints will be evaluated by various summary analyses by study visit and by change from baseline data for each efficacy endpoint. Safety and efficacy endpoints will be compared to baseline using summary statistics. Data will not be analysed by inferential statistics. | — |
Countries
Belgium, Czechia, Denmark, Italy, Norway, United Kingdom, United States
Contacts
Public ContactRossana ROCCO
Chiesi Farmaceutici S.p.A.
Outcome results
None listed