Fabry Disease
Conditions
Keywords
Glomerular filtration rate, Proteinuria, PRX-102, pegunigalsidase alfa, Fabry disease
Brief summary
The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.
Detailed description
This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.
Interventions
Recombinant human alpha galactosidase A
Sponsors
Chiesi Farmaceutici S.p.A.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Open-label extension study
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluation of treatment-related adverse events | Throughout the study, 364 weeks | CTCAE v4.03 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Kidney function 1 | Every 6 months throughout the duration of the study, 364 weeks | Estimated glomerular filtration rate (eGFRCKD-EPI) |
| Cardiac assessment | Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 | Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test |
| Biomarkers for Fabry disease | Every 6 months throughout the duration of the study, 364 weeks | Plasma Lyso-Gb3 and Gb3 |
| Kidney function 2 | Every 6 months throughout the duration of the study, 364 weeks | Protein/Creatinine ratio (UPCR), spot urine test |
| Clinical assessment | Every four weeks throughout the duration of the study, 364 weeks | Record of pain medication and pre-medication use |
| Pain assessment | Every 6 months throughout the duration of the study, 364 weeks | Short form Brief Pain Inventory (BPI) |
| Symptom assessment | Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364 | Mainz Severity Score Index (MSSI) |
| Quality of life assessment | Every 6 months throughout the duration of the study, 364 weeks | Quality of life (EQ-5D-5L) |
Countries
Belgium, Czechia, Denmark, Italy, Norway, United Kingdom, United States
Outcome results
None listed