Ignite Creation Date:
2025-12-24 @ 2:05 PM
Ignite Modification Date:
2025-12-27 @ 9:12 PM
Study NCT ID:
NCT01096095
Status:
WITHDRAWN
Last Update Posted:
2012-08-17
First Post:
2010-03-29
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D017827', 'term': 'Machado-Joseph Disease'}, {'id': 'D020754', 'term': 'Spinocerebellar Ataxias'}], 'ancestors': [{'id': 'D002524', 'term': 'Cerebellar Ataxia'}, {'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D013132', 'term': 'Spinocerebellar Degenerations'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C075773', 'term': '4-phenylbutyric acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], '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{'whyStopped': 'Regulatory authorities did not allow the entrance of the study drug in the country', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2010-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2010-05', 'completionDateStruct': {'date': '2011-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2012-08-15', 'studyFirstSubmitDate': '2010-03-29', 'studyFirstSubmitQcDate': '2010-03-29', 'lastUpdatePostDateStruct': {'date': '2012-08-17', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2010-03-30', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-12', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability of phenylbutyrate in patients with SCA3', 'timeFrame': '6 months - 12 months', 'description': 'The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).'}], 'secondaryOutcomes': [{'measure': 'Efficacy of phenylbutyrate in SCA3 on neurological dysfunction and quality of life', 'timeFrame': '6 months - 12 months', 'description': 'Efficacy outcomes are the following scores in both groups: NESSCA, SARA, 9-hole peg board test, BDI, and WHOQol.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['SCA', 'Spinocerebellar Ataxia', 'Sodium Phenylbutyrate', 'Machado-Joseph Disease', 'Treatment'], 'conditions': ['Spinocerebellar Ataxia Type 3']}, 'descriptionModule': {'briefSummary': 'DESIGN: Pilot, Phase II, double-blind, placebo-controlled study\n\nJUSTIFICATION: In the literature one does not find a pharmacological treatment that changes the natural history of Spinocerebellar ataxtia type 3 (SCA3). Patients with this disease invariably become dependent.\n\nOBJECTIVES I. To determine safety and tolerability of phenylbutyrate in patients with SCA3.\n\nII. To provide early subsidies on the efficacy of phenylbutyrate in SCA3.\n\nDURATION: 12 months of a double-blind study.\n\nPLACE OF REALIZATION: Hospital de Clínicas de Porto Alegre, Brazil.\n\nNUMBER OF PATIENTS: 20 patients.\n\nCONCOMITANT MEDICATIONS: There are no concomitant medications that are prohibited unless they affect safety parameters of this study (hemogram and platelets; fasting serum glucose, AST, ALT, Gamma-GT, Bilirubins, Prothrombin time, Creatinine, Urea, Na, K, chlorides and arterial gasometry; electrocardiogram and echocardiogram).\n\nMEDICATIONS UNDER INVESTIGATION: Powdered sodium phenylbutyrate in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets) and may be reduced in case of mild adverse events.\n\nOUTCOMES Primary safety outcome: The number of adverse events, interruptions and dose reductions in the two groups (cases and controls).\n\nEfficacy outcomes: Efficacy outcomes are the following scores in both groups: NESSCA, SARA, Barthel, BDI, and WHOQol.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '16 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. All patients shall have their molecular diagnosis confirmed and expanded polyglutamine tract (CAG) measures already determined.\n2. Patients still able to walk with until 8 years of disease duration, and\n3. Patients aged 16 years or over will be invited to participate in the study.\n\nExclusion Criteria:\n\n1. they show electrocardiogram ou echocardiographic alterations suggestive of heart insufficiency at baseline;\n2. their serum creatinine levels are higher than 1.2 mg/dL, with the confirmation of renal insufficiency due to the rate of glomerular filtration;\n3. they show a history of hypersensibility to sodium phenylbutyrate, and if\n4. they (men and women) do not agree to use a reliable contraceptive method during the entire study period and for three months after its end.'}, 'identificationModule': {'nctId': 'NCT01096095', 'briefTitle': 'Pilot Study of Safety and Efficacy of Sodium Phenylbutyrate in Spinocerebellar Ataxia Type 3', 'organization': {'class': 'OTHER', 'fullName': 'Hospital de Clinicas de Porto Alegre'}, 'officialTitle': 'Phenylbutyrate in SCA3: a Double-blind, Placebo-controlled Study to Determine Safety and Efficacy of Sodium Phenylbutyrate in Patients With SCA3', 'orgStudyIdInfo': {'id': 'CONEP CAAE 0527.0.001.000-07'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'Sodium phenylbutyrate', 'description': 'Active drug', 'interventionNames': ['Drug: Sodium Phenylbutyrate']}], 'interventions': [{'name': 'Placebo', 'type': 'DRUG', 'description': 'Powdered placebo in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets).', 'armGroupLabels': ['Placebo']}, {'name': 'Sodium Phenylbutyrate', 'type': 'DRUG', 'description': 'Powdered sodium phenylbutyrate in sachets containing each 3g. At the start of the study, the dose will be 15g/day (five sachets) and may be reduced in case of mild adverse events.', 'armGroupLabels': ['Sodium phenylbutyrate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '90.035-903', 'city': 'Porto Alegre', 'state': 'Rio Grande do Sul', 'country': 'Brazil', 'facility': 'Hospital de Clínicas de Porto Alegre', 'geoPoint': {'lat': -30.03283, 'lon': -51.23019}}], 'overallOfficials': [{'name': 'Laura B Jardim, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Medical Genetics Service of Hospital de Clínicas de Porto Alegre'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hospital de Clinicas de Porto Alegre', 'class': 'OTHER'}, 'collaborators': [{'name': 'Fundação de Amparo à Pesquisa do Estado do Rio Grande do Sul, Brazil', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}