This is the first human treatment with PRX-102, an enzyme being developed as a long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (alpha galactosidase deficiency). The safety, tolerability, and exploratory efficacy will be evaluated in this study of increasing doses. Patients will be treated with infusions every two weeks for 12 months.
Full Title of Study: “A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Months to Adult Fabry Patients”
- Study Type: Interventional
- Study Design
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Primary Purpose: Treatment
- Masking: None (Open Label)
- Study Primary Completion Date: March 6, 2016
Under the PB-102-F01 study protocol, patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) and receive PRX-102 as an intravenous infusion every 2 weeks for 12 weeks (3 months). Patients who finish the PB-102-F01 study will be enrolled in the PB-102-F02 extension study and receive the same dose they had received in the PB-102-F01 study for an additional 38 weeks (9 months).
- Drug: PRX-102
Arms, Groups and Cohorts
- Experimental: 0.2 mg/kg
- PRX-102 0.2 mg/kg every 2 weeks
- Experimental: 1 mg/kg
- PRX-102 1 mg/kg every 2 weeks
- Experimental: 2 mg/kg
- PRX-102 2 mg/kg every 2 weeks
Clinical Trial Outcome Measures
- Adverse Events
- Time Frame: 12 months
- Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG. Results represent the number of AEs that were considered possibly, probably, or definitely related to treatment.
Participating in This Clinical Trial
- Symptomatic adult Fabry patients (≥18 yrs) – Males: plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN in plasma=3.2 nmol/hr/ml, LLN in leucocytes=32 nmol/hr/mg/protein) – Females: historical genetic test results consistent with Fabry mutations – Globotriaosylceramide (Gb3) concentration in urine > 1.5 times upper normal limit – Patients who have never received enzyme replacement therapy (ERT) in the past, or patients who have not received ERT in the past 6 months and have a negative anti alpha galactosidase antibody test – eGFR ≥ 60 mL/min/1.73m2 – The patient signs informed consent – Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically acceptable method of contraception, not including the rhythm method Exclusion Criteria:
- Participation in any trial of an investigational drug within 30 days prior to study screening – Chronic kidney disease stages 3-5 (CKD 3-5) (Appendix 7) – History of dialysis or renal transplantation – Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated or dose changed in the 4 weeks prior to screening – Severe myocardial fibrosis by MRI (≥2 late-enhancement [LE] positive left ventricular segments) (Weidemann et al. 2009) – History of clinical stroke – Pregnant or nursing – Presence of HIV and/or HBsAg and/or Hepatitis C infections – Known allergies to ERT – Known allergy to Gadolinium based contrast agents – Presence of any medical, emotional, behavioral or psychological condition that, in the judgment of the Investigator and/or Medical Director, would interfere with the patient's compliance with the requirements of the study
Gender Eligibility: All
Minimum Age: 18 Years
Maximum Age: N/A
Are Healthy Volunteers Accepted: No
- Lead Sponsor
- Provider of Information About this Clinical Study
- Overall Official(s)
- Einat Almon, PhD, Study Director, Protalix
Citations Reporting on Results
Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: A 1-year Phase 1/2 clinical trial. J Inherit Metab Dis. 2019 May;42(3):534-544. doi: 10.1002/jimd.12080. Epub 2019 Apr 8.
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