Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01678898




Registration number
NCT01678898
Ethics application status
Date submitted
31/08/2012
Date registered
5/09/2012
Date last updated
13/09/2023

Titles & IDs
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
Secondary ID [1] 0 0
PB-102-F01 & 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
Experimental: 0.2 mg/kg - PRX-102 0.2 mg/kg every 2 weeks

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

Experimental: 2 mg/kg - PRX-102 2 mg/kg every 2 weeks
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse Events
Timepoint [1] 0 0
12 months

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

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
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
1/10/2012
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)
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Victoria Park
Recruitment postcode(s) [1] 0 0
3050 - Victoria Park
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
United States of America
State/province [9] 0 0
Virginia
Country [10] 0 0
Paraguay
State/province [10] 0 0
Asuncion
Country [11] 0 0
Serbia
State/province [11] 0 0
Belgrade
Country [12] 0 0
Spain
State/province [12] 0 0
Zaragoza
Country [13] 0 0
United Kingdom
State/province [13] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Protalix
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Chiesi Farmaceutici S.p.A.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

TypeCitations or Other Details
Journal Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, ... [More Details]

Results are available at https://clinicaltrials.gov/study/NCT01678898