Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
Study Identifier:
PB-102-F01 & PB-102-F02
ClinicalTrials.gov Identifier:
EudraCT Identifier:
N/A
EU CT ID:
N/A
Study Contact Information:
N/A
Study Complete
Study Details
Medical Condition
- Fabry Disease
Study Drug
- Drug: PRX-102
Date
Oct 2012 - Mar 2016
Patient Requirements
Sex: Female & Male
Age: 18+ years
Requirements Information
Inclusion and 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
Healthy Volunteers
No
Protocol Summary
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.
Study Locations
Location
Status
Location
UC Davis Medical Center, MIND Institute Department of Pediatrics, Section of Genetics
Sacramento, California, United States, 95817
Status
N/A
Location
Department of Human Genetics, Emory University School of Medicine
Atlanta, Georgia, United States, 30322
Status
N/A
Location
University of Iowa Health Clinics
Iowa City, Iowa, United States, 52242
Status
N/A
Location
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
Status
N/A
Location
Johns Hopkins University School of Medicine
Baltimore, Maryland, United States, 21205
Status
N/A
Location
Duke University Medical Center
Durham, North Carolina, United States, 27710
Status
N/A
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