Ignite Creation Date:
2025-12-17 @ 1:41 PM
Ignite Modification Date:
2025-12-23 @ 10:24 PM
Study NCT ID:
NCT01432561
Status:
COMPLETED
Last Update Posted:
2013-10-09
First Post:
2011-09-09
Is Possible Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-22'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003554', 'term': 'Cystinosis'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}], 'ancestors': [{'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D003543', 'term': 'Cysteamine'}], 'ancestors': [{'id': 'D008624', 'term': 'Mercaptoethylamines'}, {'id': 'D005021', 'term': 'Ethylamines'}, {'id': 'D000588', 'term': 'Amines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D013438', 'term': 'Sulfhydryl Compounds'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'rdohil@ucsd.edu', 'phone': '619-471-9554', 'title': 'Dr. Ranjan Dohil, P.I.', 'organization': 'University of California San Diego'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': False}}, 'adverseEventsModule': {'timeFrame': 'Adverse event data collected beginning date of enrollment and continuing to study withdrawal/termination or up to 7 days after last study visit, as applicable. Total duration of data collection varied among the subjects based on clinic scheduling.', 'description': "At study visits, subjects queried at specified intervals before and after drug administration (t=0, 30, 60, 90, 120 and 180 minutes) via a brief survey. Spontaneous A.E. observed and noted also.\n\nBetween study visits, A.E.'s as reported by participants at the beginning of each study visit and 7 days after the final study visit were noted .", 'eventGroups': [{'id': 'EG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate, 500mg once a day, three days.\n\nCysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4 which must occur within a 14 day period.', 'otherNumAtRisk': 8, 'otherNumAffected': 3, 'seriousNumAtRisk': 8, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 3, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 8, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4'}], 'classes': [{'title': 'Fasted', 'categories': [{'measurements': [{'value': '3618', 'spread': '372', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Fat/Calorie', 'categories': [{'measurements': [{'value': '2799', 'spread': '405', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Protein', 'categories': [{'measurements': [{'value': '2457', 'spread': '353', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '.04', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'High-fat/calorie compared to fasted condition', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '.005', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'High-protein compared to fasted condition', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.', 'unitOfMeasure': 'min*uM', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Peak Plasma Cysteamine Concentration (Cmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4'}], 'classes': [{'title': 'Fasted', 'categories': [{'measurements': [{'value': '26.3', 'spread': '3.5', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Fat/Calorie', 'categories': [{'measurements': [{'value': '22.4', 'spread': '5.6', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Protein', 'categories': [{'measurements': [{'value': '17.2', 'spread': '2.6', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '.16', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'High-fat/calorie compared to fasted condition', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '.036', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'High-protein compared to fasted condition', 'statisticalMethod': 'ANOVA', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.', 'unitOfMeasure': 'uM', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'PRIMARY', 'title': 'Time to Peak Plasma Cysteamine Concentration (Tmax)', 'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4'}], 'classes': [{'title': 'Fasted', 'categories': [{'measurements': [{'value': '71.2', 'spread': '12.9', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Fat/Calorie', 'categories': [{'measurements': [{'value': '106.9', 'spread': '28.8', 'groupId': 'OG000'}]}]}, {'title': 'Fed High-Protein', 'categories': [{'measurements': [{'value': '120', 'spread': '23.4', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '.30', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'Fed high-fat/calorie compared to fasted condition.', 'statisticalMethod': 'Non-parametric Hodges-Lehmann method', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}, {'pValue': '.05', 'groupIds': ['OG000'], 'ciPctValue': '95', 'groupDescription': 'High-protein compared to fasted condition', 'statisticalMethod': 'Non-parametric Hodges-Lehmann method', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER', 'testedNonInferiority': False}], 'paramType': 'MEAN', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.', 'unitOfMeasure': 'minutes', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate, 500mg once a day, three days.\n\nCysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4 which must occur within a 14 day period.'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '8'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}], 'recruitmentDetails': 'Recruitment period: 09/19/2011 - 11/21/2012 Recruitment Locations: medical centers', 'preAssignmentDetails': 'Screening for liver function and H pylori infection and brief medical history in order to determine if all inclusion and exclusion criteria were met. One enrollee met all inclusion criteria but clinical lab test revealed presence of H Pylori, an exclusion factor. Two enrollees withdrew from the trial prior to assignment due to scheduling conflicts.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '8', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'Cysteamine Bitartrate', 'description': 'Cysteamine bitartrate, 500mg once a day, three days.\n\nCysteamine bitartrate : 500 mg total, single dose taken orally on visits 2, 3 \\& 4 which must occur within a 14 day period.'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '8', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age Continuous', 'classes': [{'categories': [{'measurements': [{'value': '29.9', 'spread': '8.5', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '5', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'categories': [{'measurements': [{'value': '8', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['NA'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 8}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-09', 'completionDateStruct': {'date': '2011-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2013-09-23', 'studyFirstSubmitDate': '2011-09-09', 'resultsFirstSubmitDate': '2013-07-03', 'studyFirstSubmitQcDate': '2011-09-12', 'lastUpdatePostDateStruct': {'date': '2013-10-09', 'type': 'ESTIMATED'}, 'resultsFirstSubmitQcDate': '2013-09-23', 'studyFirstPostDateStruct': {'date': '2011-09-13', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2013-10-09', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-12', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Cysteamine Absorption: Area Under the Plasma Concentration Curve (AUC)', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.'}, {'measure': 'Peak Plasma Cysteamine Concentration (Cmax)', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.'}, {'measure': 'Time to Peak Plasma Cysteamine Concentration (Tmax)', 'timeFrame': '0, 15, 30, 45, 60, 75, 90, 105, 120, 135, 150, 165, 180 minutes, and 3.5, 4, 4.5, 5, 6 hours post-dose', 'description': 'Subjects were randomized to one of two possible treatment sequences using block randomization: Sequence 1 - fasted, high-fat, high-protein or Sequence 2 - high-protein, high-fat, fasted. Sequence assignment determined the treatment condition corresponding to Period I, II \\& III visits.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Lysosomal Storage Diseases', 'Metabolic Diseases', 'Metabolism, Inborn Errors'], 'conditions': ['Cystinosis', 'Nephropathic Cystinosis']}, 'referencesModule': {'references': [{'pmid': '12110740', 'type': 'BACKGROUND', 'citation': 'Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002 Jul 11;347(2):111-21. doi: 10.1056/NEJMra020552. No abstract available.'}, {'pmid': '3550461', 'type': 'BACKGROUND', 'citation': 'Gahl WA, Reed GF, Thoene JG, Schulman JD, Rizzo WB, Jonas AJ, Denman DW, Schlesselman JJ, Corden BJ, Schneider JA. Cysteamine therapy for children with nephropathic cystinosis. N Engl J Med. 1987 Apr 16;316(16):971-7. doi: 10.1056/NEJM198704163161602.'}, {'pmid': '8455682', 'type': 'BACKGROUND', 'citation': 'Markello TC, Bernardini IM, Gahl WA. Improved renal function in children with cystinosis treated with cysteamine. N Engl J Med. 1993 Apr 22;328(16):1157-62. doi: 10.1056/NEJM199304223281604.'}, {'pmid': '3393396', 'type': 'BACKGROUND', 'citation': 'Smolin LA, Clark KF, Thoene JG, Gahl WA, Schneider JA. A comparison of the effectiveness of cysteamine and phosphocysteamine in elevating plasma cysteamine concentration and decreasing leukocyte free cystine in nephropathic cystinosis. Pediatr Res. 1988 Jun;23(6):616-20. doi: 10.1203/00006450-198806000-00018.'}]}, 'descriptionModule': {'briefSummary': "In order to meet FDA standards of safety and efficacy reporting for most new drugs, food-effect bioavailability (the impact that the presence of food in the digestive tract has on the rate and extent at which a drug is absorbed into the bloodstream and delivered to the site of action) must be collected. Cystagon™ is an FDA approved drug for the treatment of the rare disease cystinosis that became available in 1994, but there is inadequate knowledge of the food-effect on this drug's bioavailability. This study aims to investigate how food affects the absorption of Cystagon™ into the bloodstream of normal healthy adults."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Male or female, smoker (no more than 25 cigarettes daily) or non-smoker, 18 years of age and older, with BMI \\> 18 and \\< 30.0.\n2. Females of childbearing potential who are sexually active must be willing to use two forms of contraceptive methods throughout the study and for 14days after the last study drug administration.\n3. Minimum weight of 50 kg.\n4. Good health, defined as not having history of any chronic illness and not requiring any regular medication/therapy.\n5. Must swallow tablets on a regular basis.\n\nExclusion Criteria:\n\n1. Evidence of Helicobacter pylori infection, presently, or within the last year.\n2. Subjects with known hypersensitivity to cysteamine.\n3. History, currently or within the past 3 months, of the following conditions:\n\n * Pancreatitis\n * Inflammatory bowel disease\n * Malabsorption\n * Severe liver disease\n * Unstable heart disease, e.g., myocardial infarction, heart failure, arrhythmias.\n * Unstable diabetes mellitus\n * Any bleeding disorder.\n * Zollinger-Ellison syndrome\n * Malignant disease\n4. Subjects whom may be pregnant or have health issues that make it unsafe for them participate, or whose concomitant medical problems preclude them from committing to the study schedule.\n5. Use of an investigational drug within 30 days (or 90 days for biologics) prior to dosing.\n6. Use of prescription medication within 14 days prior to the first dosing;\n7. Use over-the-counter products including natural health products (e.g. food supplements and herbal supplements) within 7 days prior to the first dosing.\n8. Donation of plasma within 7 days prior to dosing. Donation or loss of blood (excluding volume drawn at screening) of 50 mL to 499 mL of blood within 30 days, or more than 499 mL within 56 days prior to dosing.\n9. Hemoglobin \\<13.5 g/dL (males) and \\<12.0 g/dL (females) and hematocrit \\<41.0% (males) and \\<36.0% (females) at screening.\n10. Breast-feeding subject.\n11. Immunization with a live attenuated vaccine 1 month prior to dosing or planned vaccination during the course of the study.\n12. Presence of fever (body temperature \\>37.6°C) (e.g. a fever associated with a symptomatic viral or bacterial infection) within 2 weeks prior to dosing.\n\n \\-'}, 'identificationModule': {'nctId': 'NCT01432561', 'briefTitle': 'Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™', 'organization': {'class': 'OTHER', 'fullName': 'University of California, San Diego'}, 'officialTitle': 'Food-Effect on Bioavailability of Cystagon™ in Normal, Healthy Adults', 'orgStudyIdInfo': {'id': '111011'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cysteamine bitartrate', 'description': 'Cysteamine bitartrate, 500mg once a day, three days.', 'interventionNames': ['Drug: Cysteamine bitartrate']}], 'interventions': [{'name': 'Cysteamine bitartrate', 'type': 'DRUG', 'otherNames': ['Cystagon', 'Cysteamine'], 'description': '500 mg total, single dose taken orally on visits 2, 3 \\& 4 which must occur within a 14 day period.', 'armGroupLabels': ['Cysteamine bitartrate']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92093', 'city': 'La Jolla', 'state': 'California', 'country': 'United States', 'facility': 'University of California, San Diego Center for Clinical Research Services (CCR)', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}], 'overallOfficials': [{'name': 'Ranjan Dohil, M.D.', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of California, San Diego'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of California, San Diego', 'class': 'OTHER'}, 'collaborators': [{'name': 'Raptor Pharmaceuticals Corp.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal Investigator', 'investigatorFullName': 'Ranjan Dohil', 'investigatorAffiliation': 'University of California, San Diego'}}}}