Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 18 September 2023
• Main ID: NCT01678898
• Date of registration: 31/08/2012
• Prospective Registration: Yes
• Primary sponsor: Protalix
• Public title: Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
• Scientific title: 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
• Date of first enrolment: October 2012
• Target sample size: 18
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT01678898
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

Australia, Paraguay, Serbia, Spain, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Drug: PRX-102

Primary Outcome(s)

Adverse Events [Time Frame: 12 months]

Secondary ID(s)

PB-102-F01 & PB-102-F02

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Chiesi Farmaceutici S.p.A.

Results

• Results available: Yes
• Date Posted: 27/01/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01678898