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Withdrawn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

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:
  • pegunigalsidase alfa
  • recombinant human alpha galactosidase-A
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

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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