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An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients

This study is enrolling participants by invitation only.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT01769001
First received: January 14, 2013
Last updated: February 6, 2013
Last verified: February 2013
History of Changes
  Purpose

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg), to receive the same dose they had received in Phase 1/2 study PB-102-F01, and will continue to receive PRX-102 as an intravenous infusion every 2 weeks for 38 weeks.


Condition Intervention Phase
Fabry Disease
 Drug: PRX-102
 Phase 1
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Extension of Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 38 Weeks to Adult Fabry Patients

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:
  • Number of participants with adverse events as a measure of safety [ Time Frame: 38 weeks ] [ Designated as safety issue: Yes ]
    Adverse events reported by the patient and from monitoring with clinical laboratory, physical examination, ECG


Secondary Outcome Measures:
  • Gb3 concentrations [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Gb3 concentrations in plasma and urine sediment

  • Glomerular filtration rate [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Measurement of glomerular filtration

  • Pain [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Short term brief pain inventory

  • Proteinuria [ Time Frame: 38 weeks ] [ Designated as safety issue: No ]
    Measure of kidney function


Estimated Enrollment: 18
Study Start Date: March 2013
Estimated Study Completion Date: September 2014
Estimated Primary Completion Date: July 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 mg/kg
PRX-102 1 mg/kg every 2 weeks
 Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
Experimental: 2 mg/kg
PRX-102 2 mg/kg every 2 weeks
 Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A
Experimental: 0.2 mg/kg
PRX-102 0.2 mg/kg every 2 weeks
 Drug: PRX-102
Comparison of different doses of drug
Other Name: plant cell expressed recombinant human alpha-galactosidase-A

Detailed Description:

Patients will be enrolled into one of three PRX-102 dosing groups (0.2 mg/kg, 1.0 mg/kg, 2.0 mg/kg) to receive the same dose they had received in the Phase 1/2 study, PB-102-F01, and will continue to receive intravenous infusions of PRX-102 every 2 weeks for 38 weeks (9 months).

All exploratory endpoints that were evaluated during the Phase 1/2 study PB-102-F01 will continue to be assessed in the extension protocol (study PB-102-F02).

The additional exploratory efficacy parameters that were evaluated at baseline of the Phase 1/2 study PB-102-F01 as a reference point, before the initiation of treatment, will be evaluated after 3 months into the extension study (total treatment of 6 months). These parameters include Gb3 concentration in renal and skin tissues, LVM, MSSI and cardiac function tests (echocardiography and stress test). Cerebrovascular disease (clinical and MRI) will be evaluated at last infusion (total treatment of 12 months).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completion of Phase 1/2 study PB-102-F01
  • 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:

  • Pregnant or nursing
  • 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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01769001

Sponsors and Collaborators
Protalix
Investigators
Study Director: Einat Almon, PhD Protalix Biotherapeutics
  More Information

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT01769001     History of Changes
Other Study ID Numbers: PB-102-F02
Study First Received: January 14, 2013
Last Updated: February 6, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by Protalix:
Fabry disease
Alpha galactosidase deficiency
Metabolic storage disease

Additional relevant MeSH terms:
Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on May 21, 2013