Ignite Creation Date:
2025-12-24 @ 11:18 PM
Ignite Modification Date:
2025-12-25 @ 8:59 PM
Study NCT ID:
NCT00067756
Status:
COMPLETED
Last Update Posted:
2014-01-03
First Post:
2003-08-26
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019896', 'term': 'alpha 1-Antitrypsin Deficiency'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D008107', 'term': 'Liver Diseases'}], 'ancestors': [{'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D013352', 'term': 'Subcutaneous Emphysema'}, {'id': 'D004646', 'term': 'Emphysema'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C121358', 'term': '4-phenylbutylamine'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 12}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2001-11'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-04', 'completionDateStruct': {'date': '2003-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2014-01-02', 'studyFirstSubmitDate': '2003-08-26', 'studyFirstSubmitQcDate': '2003-08-26', 'lastUpdatePostDateStruct': {'date': '2014-01-03', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2003-08-27', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2003-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'To determine if 4-PBA significantly increases secretion of AAT in AAT-deficient individuals with and without liver disease.', 'timeFrame': '10 days'}], 'secondaryOutcomes': [{'measure': 'To determine the pharmacokinetics of 4-PBA', 'timeFrame': '10 days'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Pulmonary Disease', 'Liver Disease'], 'conditions': ['Alpha 1-Antitrypsin Deficiency']}, 'referencesModule': {'references': [{'type': 'RESULT', 'citation': 'Gonzalez-Peralta RP, Leonard S, Harvey R, Schreck P, Vironosvkaya N, Brantly ML. 4-PHENYL BUTYRATE MEDIATED SECRETION RESCUE IN PATIENTS WITH ALPHA 1-ANTITRYPSIN (AAT) DEFICIENCY: A PILOT STUDY HEPATOLOGY, Vol. 44, No. 4, Suppl. 1, 2006 AASLD ABSTRACTS, p. 211A, 61'}], 'seeAlsoLinks': [{'url': 'http://www.alphaone.ufl.edu', 'label': 'Alpha-1 Foundation Alpha-1 Research Program'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.', 'detailedDescription': 'The purpose of this study is to determine whether 4-PBA will significantly increase serum Z AAT levels in AAT-deficient individuals with and without evidence of hepatocellular injury and to assess its effects on liver injury.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age 18-65\n* Serum A1-PI levels \\<11uM an appropriate genetic phenotype/genotype\n* 5 of 10 subjects must have documented laboratory evidence of liver disease\n* Willingness to withhold Prolastin therapy for 6 weeks prior to screening and throughout the 4-PBA dosing period (up to 3 months)\n\nExclusion Criteria:\n\n* Any cause of liver disease other than Alpha-1 Antitrypsin deficiency\n* Evidence of advanced liver disease\n* HIV positive\n* Use of systemic steroids, ursodeoxycholic acid (Actigall, Urso), or herbs in the prior 6 months'}, 'identificationModule': {'nctId': 'NCT00067756', 'briefTitle': '4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?', 'organization': {'class': 'OTHER', 'fullName': 'University of Florida'}, 'officialTitle': '"4 Phenyl Butyrate Mediated Secretion Rescue in Alpha 1-Antitrypsin Deficient Individuals"', 'orgStudyIdInfo': {'id': '87-2001'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '4-PBA', 'description': 'The study will involve a 4-PBA dose escalation and pharmacokinetics component The study group will be comprised of a total of at least 10 AAT-deficient,(phenotype ZZ referred to as PiZZ) patients. These patients will be divided into two groups: with and without clinical evidence of mild to moderate hepatocellular injury.', 'interventionNames': ['Drug: 4-PBA']}], 'interventions': [{'name': '4-PBA', 'type': 'DRUG', 'otherNames': ['4-phenyl butyric acid'], 'description': 'During the first 3 days of this phase baseline serum AAT levels will be determined. The participants will be then given increased amounts of 4-PBA orally in 6 divided doses (day 4-6, 30 g/day and day 7-9, 40/day', 'armGroupLabels': ['4-PBA']}]}, 'contactsLocationsModule': {'locations': [{'zip': '32610', 'city': 'Gainesville', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Florida', 'geoPoint': {'lat': 29.65163, 'lon': -82.32483}}], 'overallOfficials': [{'name': 'Mark L Brantly, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Florida'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Florida', 'class': 'OTHER'}, 'collaborators': [{'name': 'Alpha-1 Foundation', 'class': 'OTHER'}, {'name': 'Brantly, Mark L., M.D.', 'class': 'INDIV'}], 'responsibleParty': {'type': 'SPONSOR'}}}}