Ignite Creation Date:
2025-12-24 @ 7:16 PM
Ignite Modification Date:
2026-01-02 @ 3:01 PM
Study NCT ID:
NCT00640003
Status:
COMPLETED
Last Update Posted:
2017-12-20
First Post:
2008-03-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Baclofen Treatment of Ataxia Telangiectasia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D001260', 'term': 'Ataxia Telangiectasia'}, {'id': 'D001259', 'term': 'Ataxia'}, {'id': 'D013684', 'term': 'Telangiectasis'}], 'ancestors': [{'id': 'D020754', 'term': 'Spinocerebellar Ataxias'}, {'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': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D020820', 'term': 'Dyskinesias'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D000081207', 'term': 'Primary Immunodeficiency Diseases'}, {'id': 'D049914', 'term': 'DNA Repair-Deficiency Disorders'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D007153', 'term': 'Immunologic Deficiency Syndromes'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D001418', 'term': 'Baclofen'}, {'id': 'D020148', 'term': 'Butyric Acid'}], 'ancestors': [{'id': 'D005680', 'term': 'gamma-Aminobutyric Acid'}, {'id': 'D000613', 'term': 'Aminobutyrates'}, {'id': 'D002087', 'term': 'Butyrates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D005232', 'term': 'Fatty Acids, Volatile'}, {'id': 'D005227', 'term': 'Fatty Acids'}, {'id': 'D008055', 'term': 'Lipids'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2007-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2011-02', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-18', 'studyFirstSubmitDate': '2008-03-14', 'studyFirstSubmitQcDate': '2008-03-19', 'lastUpdatePostDateStruct': {'date': '2017-12-20', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2008-03-20', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-02', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Improvement in the decay constant for velocity storage as assessed by quantitative video examination of eye movement during and after rotation.', 'timeFrame': '3 months'}], 'secondaryOutcomes': [{'measure': 'Other outcome measures include quantitative measurement of tremor, postural stability and a standard neurologic examination.', 'timeFrame': '3 months'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['AT', 'Ataxia', 'Telangiectasia'], 'conditions': ['Ataxia Telangiectasia']}, 'descriptionModule': {'briefSummary': 'This research is being done to find out if Baclofen, a medicine that is often used for the treatment of abnormal stiffness, might also be useful to treat some of the neurologic problems caused by ataxia telangiectasia (A-T). The investigators also want to find out if there are better ways to measure the problems of ataxia and abnormal eye movement for future studies of medication in ataxia telangiectasia.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Teenagers over 12 years old and young adults with A-T may join if they have been evaluated previously in the A-T Clinical Center at Johns Hopkins Hospital and have a measurable abnormality of eye movement.\n* Patients who are presently taking Baclofen will be eligible for the study if they are presently receiving the medication under the direction of Dr. Crawford in the ATCC, and are willing to withdraw from the medication for a period of one month prior to the initial screening visit.\n* Female patients who are sexually active will be given a standard serum HCG pregnancy test.\n* Those who are sexually active will be counseled about necessary precautions against pregnancy during the duration of the trial.\n\nExclusion Criteria:\n\n* A positive pregnancy test.'}, 'identificationModule': {'nctId': 'NCT00640003', 'briefTitle': 'Baclofen Treatment of Ataxia Telangiectasia', 'organization': {'class': 'OTHER', 'fullName': 'Johns Hopkins University'}, 'orgStudyIdInfo': {'id': 'NA_00002180'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '1', 'interventionNames': ['Drug: Baclofen', 'Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': '2', 'interventionNames': ['Drug: Baclofen', 'Drug: Placebo']}], 'interventions': [{'name': 'Baclofen', 'type': 'DRUG', 'otherNames': ['Butanoic Acid'], 'description': 'Drug given by orally via capsules. Escalation steps in 4-day intervals. Step 1: 5mg QAM. Step 2: 5mg BID. Step 3: 5mg TID. Step 4: 10mg QAM, 5mg BID. Step 5: 10mg BID, 5mg QMid-day. Step 6: 10mg TID. Step 6 will last up to 2 weeks. De-escalation will start from highest dose step and work backwards in 2-day intervals. Complete drug administration of escalating, staying constant at 10mg TID and de-escalating will be done with both drug and placebo.', 'armGroupLabels': ['1', '2']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'placebo drug', 'armGroupLabels': ['1', '2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '21108', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins Hospital', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins Hospital', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}], 'overallOfficials': [{'name': 'Thomas O Crawford, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Johns Hopkins School of Medicine'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Johns Hopkins University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}