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

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Protalix
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01678898
First received: August 31, 2012
Last updated: April 4, 2014
Last verified: April 2014

August 31, 2012
April 4, 2014
October 2012
December 2014   (final data collection date for primary outcome measure)
Adverse events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
Reportings of adverse events reported by the patient and from monitoring with clinical laboratory, physical examination and ECG
Same as current
Complete list of historical versions of study NCT01678898 on ClinicalTrials.gov Archive Site
  • Gb3 concentrations [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Gb3 concentrations in plasma and urine sediment
  • Kidney function [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
    Measurement of glomerular filtration and proteinuria
  • Pain [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
    Short term brief pain inventory
Same as current
Not Provided
Not Provided
 
Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
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 Weeks to Adult Fabry Patients

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 weeks (7 infusions).

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Fabry Disease
Drug: PRX-102
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
  • Experimental: 0.2 mg/kg
    PRX-102 0.2 mg/kg every 2 weeks
    Intervention: Drug: PRX-102
  • Experimental: 1 mg/kg
    PRX-102 1 mg/kg every 2 weeks
    Intervention: Drug: PRX-102
  • Experimental: 2 mg/kg
    PRX-102 2 mg/kg every 2 weeks
    Intervention: Drug: PRX-102
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
18
January 2015
December 2014   (final data collection date for primary outcome measure)

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
  • Chronic kidney disease - stages 1 or 2 (CKD1 or 2) (Appendix 7) with proteinuria > 200 mg/g protein-to-creatinine ratio or equivalent, measured in a Spot urine sample, demonstrated in at least one of 2 separate samples (1 sample at screening visit and the other from historical data)
  • 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
Both
18 Years and older
No
United States,   Australia,   Paraguay,   United Kingdom
 
NCT01678898
PB-102-F01
No
Protalix
Protalix
Not Provided
Study Director: Einat Almon, PhD Protalix
Protalix
April 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP