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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01769001




Registration number
NCT01769001
Ethics application status
Date submitted
14/01/2013
Date registered
16/01/2013
Date last updated
11/01/2018

Titles & IDs
Public title
An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients
Scientific 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
Secondary ID [1] 0 0
PB-102-F02
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PRX-102

Experimental: 1 mg/kg - PRX-102 1 mg/kg every 2 weeks

Experimental: 2 mg/kg - PRX-102 2 mg/kg every 2 weeks

Experimental: 0.2 mg/kg - PRX-102 0.2 mg/kg every 2 weeks


Treatment: Drugs: PRX-102
Comparison of different doses of drug
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of participants with adverse events as a measure of safety
Timepoint [1] 0 0
38 weeks
Secondary outcome [1] 0 0
Gb3 concentrations
Timepoint [1] 0 0
38 weeks
Secondary outcome [2] 0 0
Glomerular filtration rate
Timepoint [2] 0 0
38 weeks
Secondary outcome [3] 0 0
Pain
Timepoint [3] 0 0
38 weeks
Secondary outcome [4] 0 0
Proteinuria
Timepoint [4] 0 0
38 weeks

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 1/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/03/2013
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
6/03/2016
Sample size
Target
Accrual to date
Final
18
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3050 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Iowa
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
North Carolina
Country [6] 0 0
United States of America
State/province [6] 0 0
Texas
Country [7] 0 0
United States of America
State/province [7] 0 0
Virginia
Country [8] 0 0
Paraguay
State/province [8] 0 0
Asuncion
Country [9] 0 0
Spain
State/province [9] 0 0
Zaragoza
Country [10] 0 0
United Kingdom
State/province [10] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Protalix
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/show/NCT01769001
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Einat Almon, PhD
Address 0 0
Protalix Biotherapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/show/NCT01769001