Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02921620
• Date of registration: 26/09/2016
• Prospective Registration: Yes
• Primary sponsor: Protalix
• Public title: Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
• Scientific title: A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients
• Date of first enrolment: July 2017
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT02921620
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Contacts

• Name: Raul Chertkoff, MD
• Affiliation: Protalix Ltd.

Key inclusion & exclusion 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



Age minimum: 14 Years
Age maximum: 45 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Fabry Disease

Intervention(s)

Biological: PRX-102

Other: Placebo

Primary Outcome(s)

IBSSS Part 1 [Time Frame: Every 2 weeks for 6 months]

Secondary Outcome(s)

Plasma Gb3 [Time Frame: Every 4 weeks for 6 months]

Plasma Lyso-Gb3 [Time Frame: Every 4 weeks for 6 months]

Stool frequency [Time Frame: After every bowel movement for 6 months]

Frequency of pain medication use [Time Frame: Every 2 weeks for 6 months]

Body Weight [Time Frame: Every 2 weeks for 6 months]

Urine Lyso-GB3 [Time Frame: Every 6 weeks for 6 months]

Secondary ID(s)

PB-102-F10

Source(s) of Monetary Support

Please refer to primary and secondary sponsors