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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02921620
Recruitment Status : Withdrawn (Protocol not feasible)
First Posted : October 3, 2016
Last Update Posted : January 8, 2018
Sponsor:
Information provided by (Responsible Party):
Protalix

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Fabry Disease Biological: PRX-102 Other: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Estimated Study Start Date : July 2017
Estimated Primary Completion Date : July 2018
Estimated Study Completion Date : July 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: PRX-102
PRX-102 infusions every 2 weeks
Biological: PRX-102
Intravenous
Other Names:
  • pegunigalsidase alfa
  • recombinant human alpha galactosidase-A

Placebo Comparator: Placebo
Placebo infusions every 2 weeks
Other: Placebo
Intravenous




Primary Outcome Measures :
  1. IBSSS Part 1 [ Time Frame: Every 2 weeks for 6 months ]
    Irritable Bowel Syndrome Severity Score


Secondary Outcome Measures :
  1. Stool frequency [ Time Frame: After every bowel movement for 6 months ]
    from BSFS diary

  2. Body Weight [ Time Frame: Every 2 weeks for 6 months ]
  3. Plasma Lyso-Gb3 [ Time Frame: Every 4 weeks for 6 months ]
  4. Plasma Gb3 [ Time Frame: Every 4 weeks for 6 months ]
  5. Urine Lyso-GB3 [ Time Frame: Every 6 weeks for 6 months ]
  6. Frequency of pain medication use [ Time Frame: Every 2 weeks for 6 months ]

Other Outcome Measures:
  1. Anti-PRX-102 antibodies [ Time Frame: Every 4 weeks for 6 months ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   14 Years to 45 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02921620


Sponsors and Collaborators
Protalix
Investigators
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Study Director: Raul Chertkoff, MD Protalix Ltd

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Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT02921620     History of Changes
Other Study ID Numbers: PB-102-F10
First Posted: October 3, 2016    Key Record Dates
Last Update Posted: January 8, 2018
Last Verified: January 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Protalix:
Gastrointestinal symptoms
Diarrhea
Abdominal pain

Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders