Eligibility Module
The Eligibility Module contains detailed information about who can participate in the clinical trial. This includes eligibility criteria, age restrictions, gender requirements, healthy volunteer status, and study population descriptions, helping researchers understand who is eligible to participate in the study.
Eligibility Module path is as follows:
Study -> Protocol Section -> Eligibility Module
Eligibility Module
Ignite Creation Date:
2025-12-25 @ 4:06 AM
Ignite Modification Date:
2025-12-25 @ 4:06 AM
NCT ID:
NCT02921620
Eligibility Criteria:
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
Healthy Volunteers:
False
Sex:
MALE
Minimum Age:
14 Years
Maximum Age:
45 Years
Study:
NCT02921620
Study Brief:
Protocol Section:
NCT02921620