Ignite Creation Date:
2025-12-25 @ 4:06 AM
Ignite Modification Date:
2026-02-25 @ 5:53 PM
Study NCT ID:
NCT02921620
Status:
WITHDRAWN
Last Update Posted:
2018-01-08
First Post:
2016-09-26
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D003967', 'term': 'Diarrhea'}, {'id': 'D015746', 'term': 'Abdominal Pain'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D012817', 'term': 'Signs and Symptoms, Digestive'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D010146', 'term': 'Pain'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'Protocol not feasible', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2017-07', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-01', 'completionDateStruct': {'date': '2018-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2018-01-04', 'studyFirstSubmitDate': '2016-09-26', 'studyFirstSubmitQcDate': '2016-09-29', 'lastUpdatePostDateStruct': {'date': '2018-01-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2016-10-03', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2018-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Anti-PRX-102 antibodies', 'timeFrame': 'Every 4 weeks for 6 months'}], 'primaryOutcomes': [{'measure': 'IBSSS Part 1', 'timeFrame': 'Every 2 weeks for 6 months', 'description': 'Irritable Bowel Syndrome Severity Score'}], 'secondaryOutcomes': [{'measure': 'Stool frequency', 'timeFrame': 'After every bowel movement for 6 months', 'description': 'from BSFS diary'}, {'measure': 'Body Weight', 'timeFrame': 'Every 2 weeks for 6 months'}, {'measure': 'Plasma Lyso-Gb3', 'timeFrame': 'Every 4 weeks for 6 months'}, {'measure': 'Plasma Gb3', 'timeFrame': 'Every 4 weeks for 6 months'}, {'measure': 'Urine Lyso-GB3', 'timeFrame': 'Every 6 weeks for 6 months'}, {'measure': 'Frequency of pain medication use', 'timeFrame': 'Every 2 weeks for 6 months'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Gastrointestinal symptoms', 'Diarrhea', 'Abdominal pain'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': '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.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '45 Years', 'minimumAge': '14 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* 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\n* A documented diagnosis of Fabry disease: Plasma and/or leucocyte alpha galactosidase activity (by activity assay) less than lower limit of normal (LLN)\n* eGFR by CKD-EPI \\> 30 ml/min/1.73 m2\n* Moderate to severe gastrointestinal symptoms as defined by:\n\n * Average score of \\> 175 from at least two Irritable Bowel Symptom Severity Score (IBSSS) Part 1 assessments before randomization.\n * 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\n * ≥ 3 stools a day with a consistency of ≥ 5 on the BSFS during the week before randomization.\n* 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.\n\nExclusion Criteria:\n\n* Patients will be evaluated to rule out other gastrointestinal comorbidity than Fabry disease as responsible for the gastrointestinal symptoms by:\n\n 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\n* Use of any kind of laxatives\n* Initiation of anti-diarrheal medications during the screening period\n* History of renal dialysis or transplantation\n* 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\n* Cardiovascular event (myocardial infarction, unstable angina) in the 6 month period before randomization\n* Congestive heart failure NYHA Class IV\n* Cerebrovascular event (stroke, transient ischemic attack) in the 6 month period before randomization\n* Known history of hypersensitivity to Gadolinium contrast agent\n* Known allergies to ERT\n* 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"}, 'identificationModule': {'nctId': 'NCT02921620', 'briefTitle': 'Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'Protalix'}, 'officialTitle': 'A Randomized, Double Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease Patients', 'orgStudyIdInfo': {'id': 'PB-102-F10'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PRX-102', 'description': 'PRX-102 infusions every 2 weeks', 'interventionNames': ['Biological: PRX-102']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo infusions every 2 weeks', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'PRX-102', 'type': 'BIOLOGICAL', 'otherNames': ['pegunigalsidase alfa', 'recombinant human alpha galactosidase-A'], 'description': 'Intravenous', 'armGroupLabels': ['PRX-102']}, {'name': 'Placebo', 'type': 'OTHER', 'description': 'Intravenous', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'overallOfficials': [{'name': 'Raul Chertkoff, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Protalix Ltd.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Protalix', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}