- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02921620
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
January 4, 2018 updated by: Protalix
A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients
The study will be a randomized, double blind, placebo-controlled study of the safety and efficacy of PRX-102 in ERT naïve male patients randomized 1:1.
Patient age will be 14 to 45 years.
Patients must have diarrhea defined as ≥ 3 stools a day with an average consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient electronic diary and moderate to severe gastrointestinal symptoms as defined by the Irritable Bowel Symptom Severity Score (IBSSS) Part 1 average > 175 derived from at least two IBSSS assessments during screening period.
Patients will receive intravenous infusions of PRX-102 1 mg/kg or placebo every two weeks for 6 months.
Study Overview
Status
Withdrawn
Conditions
Intervention / Treatment
Study Type
Interventional
Phase
- Phase 3
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
14 years to 45 years (Child, Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
Inclusion Criteria:
- Males, age 14-45 years, naïve to enzyme replacement therapy (ERT) or off ERT or off chaperone treatment for at least 6 months and negative for anti-PRX-102 antibodies
- A documented diagnosis of Fabry disease: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN)
- eGFR by CKD-EPI > 30 ml/min/1.73 m2
Moderate to severe gastrointestinal symptoms as defined by:
- Average score of > 175 from at least two Irritable Bowel Symptom Severity Score (IBSSS) Part 1 assessments before randomization.
- Average stool consistency of ≥ 5.5 on the Bristol Stool Form Scale (BSFS) by patient diary during 2 weeks prior to randomization out of the 4 week of screening period and
- ≥ 3 stools a day with a consistency of ≥ 5 on the BSFS during the week before randomization.
- Completed electronic BSFS diary on at least 6 of the 7 days during the week prior to randomization AND at least 11 of the 14 days during the 2 weeks prior to randomization.
Exclusion Criteria:
Patients will be evaluated to rule out other gastrointestinal comorbidity than Fabry disease as responsible for the gastrointestinal symptoms by:
i. Medical History for non Fabry gastrointestinal comorbidity ii. Occult blood in stool iii. Stool culture for bacteria and parasites iv. Calprotectin in stool v. Sigmoidoscopy
- Use of any kind of laxatives
- Initiation of anti-diarrheal medications during the screening period
- History of renal dialysis or transplantation
- Use of, or change in dose of, angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) for less than 4 weeks prior to screening
- Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization
- Congestive heart failure NYHA Class IV
- Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization
- Known history of hypersensitivity to Gadolinium contrast agent
- Known allergies to ERT
- 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 Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: PRX-102
PRX-102 infusions every 2 weeks
|
Intravenous
Other Names:
|
Placebo Comparator: Placebo
Placebo infusions every 2 weeks
|
Intravenous
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
IBSSS Part 1
Time Frame: Every 2 weeks for 6 months
|
Irritable Bowel Syndrome Severity Score
|
Every 2 weeks for 6 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Stool frequency
Time Frame: After every bowel movement for 6 months
|
from BSFS diary
|
After every bowel movement for 6 months
|
Body Weight
Time Frame: Every 2 weeks for 6 months
|
Every 2 weeks for 6 months
|
|
Plasma Lyso-Gb3
Time Frame: Every 4 weeks for 6 months
|
Every 4 weeks for 6 months
|
|
Plasma Gb3
Time Frame: Every 4 weeks for 6 months
|
Every 4 weeks for 6 months
|
|
Urine Lyso-GB3
Time Frame: Every 6 weeks for 6 months
|
Every 6 weeks for 6 months
|
|
Frequency of pain medication use
Time Frame: Every 2 weeks for 6 months
|
Every 2 weeks for 6 months
|
Other Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Anti-PRX-102 antibodies
Time Frame: Every 4 weeks for 6 months
|
Every 4 weeks for 6 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Anticipated)
July 1, 2017
Primary Completion (Anticipated)
July 1, 2018
Study Completion (Anticipated)
July 1, 2018
Study Registration Dates
First Submitted
September 26, 2016
First Submitted That Met QC Criteria
September 29, 2016
First Posted (Estimate)
October 3, 2016
Study Record Updates
Last Update Posted (Actual)
January 8, 2018
Last Update Submitted That Met QC Criteria
January 4, 2018
Last Verified
January 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Metabolic Diseases
- Cerebrovascular Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Cerebral Small Vessel Diseases
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Fabry Disease
Other Study ID Numbers
- PB-102-F10
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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