Ignite Creation Date:
2025-12-24 @ 12:21 PM
Ignite Modification Date:
2026-03-01 @ 6:11 AM
Study NCT ID:
NCT01000961
Status:
COMPLETED
Last Update Posted:
2024-12-19
First Post:
2009-10-22
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003554', 'term': 'Cystinosis'}, {'id': 'D035583', 'term': 'Rare Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}], 'ancestors': [{'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D020969', 'term': 'Disease Attributes'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}, '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': 'clinicaltrials@horizonpharma.com', 'phone': '224-383-3000', 'title': 'Evelyn Olson, Director', 'organization': 'Horizon Pharma USA, Inc.'}, 'certainAgreement': {'otherDetails': 'Investigators may publish or disclose study data, with the restriction that sponsor may embargo such communications for a period up to 60 days from the time submitted to sponsor. As a multi-center study, first publication of results will take place in conjunction with all investigators. If such a multi-center publication is not forthcoming within 18 months of study completion, individual investigators may publish as restricted according to limitations expressed above (60 day embargo).', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Treatment periods', 'description': 'Safety population AE reporting', 'eventGroups': [{'id': 'EG000', 'title': 'RP103', 'description': 'Safety Population during treatment periods', 'otherNumAtRisk': 43, 'otherNumAffected': 16, 'seriousNumAtRisk': 43, 'seriousNumAffected': 6}, {'id': 'EG001', 'title': 'Cystagon®', 'description': 'Safety population during treatment periods', 'otherNumAtRisk': 41, 'otherNumAffected': 5, 'seriousNumAtRisk': 41, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 8, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 5, 'numAffected': 3}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 7, 'numAffected': 5}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 3, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Abdominal Pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 4, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 3, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}], 'seriousEvents': [{'term': 'Knee deformity', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Hypovolaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 0, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 1, 'numAffected': 1}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Abdominal Discomfort', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Hypokalaemia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Metabolism and nutrition disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}, {'term': 'Femur Fracture', 'stats': [{'groupId': 'EG000', 'numAtRisk': 43, 'numEvents': 1, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 41, 'numEvents': 0, 'numAffected': 0}], 'organSystem': 'Injury, poisoning and procedural complications', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '39', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'RP103', 'description': 'Per Protocol Population'}, {'id': 'OG001', 'title': 'Cystagon®', 'description': 'Per Protocol Population'}], 'classes': [{'categories': [{'measurements': [{'value': '0.5152', 'spread': '0.05555', 'groupId': 'OG000'}, {'value': '0.4367', 'spread': '0.05555', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0001', 'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95.8', 'paramValue': '0.0785', 'ciLowerLimit': '0.0107', 'ciUpperLimit': '0.1464', 'estimateComment': '95.8% confidence interval was used instead of 95% to take into account a sample size re-estimation calculation that was performed after 20 patients were enrolled.', 'groupDescription': '16-subject study will have 90% power to reject the null hypothesis of non-inferiority at the 0.025 level of significance with a non-inferiority margin of 0.3. Final analysis was performed at a nominal significance level of 0.02104.', 'statisticalMethod': 't-test, 1 sided', 'nonInferiorityType': 'NON_INFERIORITY_OR_EQUIVALENCE_LEGACY', 'nonInferiorityComment': 'The non-inferiority endpoint of the clinical trial would be achieved if the upper limit of the 95.8% CI of the difference between RP103 and Cystagon® was less than the a-priori 0.3 non-inferiority margin, which would correspond to an observed p-value less than or equal to 0.02104'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '4 weeks after the last subject has completed the study', 'unitOfMeasure': 'nmol ½ Cystine / mg protein', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cystagon®', 'description': 'Per Protocol Population'}, {'id': 'OG001', 'title': 'RP103', 'description': 'Per Protocol Population'}], 'classes': [{'categories': [{'measurements': [{'value': '2.73', 'spread': '1.36', 'groupId': 'OG000'}, {'value': '3.70', 'spread': '1.72', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Median Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '1.40', 'ciLowerLimit': '1.17', 'ciUpperLimit': '1.67', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '4 weeks after the last subject has completed the study', 'unitOfMeasure': 'Cmax (mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cystagon®', 'description': 'Per Protocol Population'}, {'id': 'OG001', 'title': 'RP103', 'description': 'Per Protocol Population'}], 'classes': [{'categories': [{'measurements': [{'value': '72', 'spread': '31', 'groupId': 'OG000'}, {'value': '187', 'spread': '89', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '105', 'ciLowerLimit': '90', 'ciUpperLimit': '150', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '4 weeks after the last subject has completed the study', 'unitOfMeasure': 'Tmax (minute)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®.', 'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'OG000'}, {'value': '37', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Cystagon®', 'description': 'Per Protocol Population'}, {'id': 'OG001', 'title': 'RP103', 'description': 'Per Protocol Population'}], 'classes': [{'categories': [{'measurements': [{'value': '357', 'spread': '150', 'groupId': 'OG000'}, {'value': '739', 'spread': '334', 'groupId': 'OG001'}]}]}], 'analyses': [{'groupIds': ['OG000', 'OG001'], 'paramType': 'Mean Difference (Final Values)', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '2.03', 'ciLowerLimit': '1.75', 'ciUpperLimit': '2.39', 'nonInferiorityType': 'SUPERIORITY_OR_OTHER_LEGACY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': '6 hours post dosing for Cystagon®; 12 hours post dosing for RP103.', 'unitOfMeasure': 'AUC(0-t) (min*mg/L)', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Cystagon First, Then Cystagon, Then RP103', 'description': 'Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in first intervention (after Run-in period) and RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in second intervention period.'}, {'id': 'FG001', 'title': 'Cystagon First, Then RP103, Then Cystagon', 'description': 'RP103 dose in 25 mg and 75 mg capsule formulations administered every 12 hours in first intervention (after Run-in period) and Cystagon® dose in 50 mg and 150 mg capsule formulations administered every 6 hours in second intervention period.'}], 'periods': [{'title': 'Run-in Period of 2-3 Weeks on Cystagon', 'milestones': [{'type': 'STARTED', 'achievements': [{'comment': 'Part of the Safety analysis set', 'groupId': 'FG000', 'numSubjects': '21'}, {'comment': 'Part of the Safety analysis set', 'groupId': 'FG001', 'numSubjects': '22'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '22'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}, {'title': 'First Intervention (3 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '22'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Infection after pre-planned surgery', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '1'}]}]}, {'title': 'Second Intervention (3 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'COMPLETED', 'achievements': [{'comment': 'Part of Efficacy analysis set', 'groupId': 'FG000', 'numSubjects': '21'}, {'comment': 'Part of Efficacy analysis set', 'groupId': 'FG001', 'numSubjects': '20'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '0'}]}]}], 'preAssignmentDetails': 'Participants randomized to each per sequence Arm are expected to remain in the same Arm throughout all intervention periods.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '39', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': 'RP103 and Cystagon® Crossover', 'description': 'Per Protocol Population'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '36', 'groupId': 'BG000'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '3', 'groupId': 'BG000'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '11.9', 'spread': '4.33', 'groupId': 'BG000'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '16', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '23', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'France', 'categories': [{'measurements': [{'value': '13', 'groupId': 'BG000'}]}]}, {'title': 'United States', 'categories': [{'measurements': [{'value': '22', 'groupId': 'BG000'}]}]}, {'title': 'Netherlands', 'categories': [{'measurements': [{'value': '4', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}], 'populationDescription': 'There were 39 participants analyzed for Cystagon®; and there were 37 participants analyzed for RP103.'}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'CROSSOVER'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 43}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-06'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-11', 'completionDateStruct': {'date': '2011-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2024-11-25', 'studyFirstSubmitDate': '2009-10-22', 'resultsFirstSubmitDate': '2012-11-02', 'studyFirstSubmitQcDate': '2009-10-22', 'lastUpdatePostDateStruct': {'date': '2024-12-19', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2014-11-18', 'studyFirstPostDateStruct': {'date': '2009-10-23', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2014-11-19', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-06', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'The Steady-state White Blood Cell Cystine Levels of RP103 Compared to Cystagon®', 'timeFrame': '4 weeks after the last subject has completed the study'}], 'secondaryOutcomes': [{'measure': 'Comparison of Cysteamine PK Profiles, Steady State Cmax, Between RP103 and Cystagon®.', 'timeFrame': '4 weeks after the last subject has completed the study'}, {'measure': 'Comparison of Cysteamine PK Profiles, Steady State Tmax, Between RP103 and Cystagon®.', 'timeFrame': '4 weeks after the last subject has completed the study'}, {'measure': 'Comparison of Cysteamine PK Profiles, AUC(0-t), Between RP103 and Cystagon®.', 'timeFrame': '6 hours post dosing for Cystagon®; 12 hours post dosing for RP103.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['cystinosis', 'cysteamine', 'inheritable disease', 'orphan disease', 'CTNS protein, human', 'metabolic disease', 'nephropathic cystinosis'], 'conditions': ['Cystinosis']}, 'referencesModule': {'references': [{'pmid': '16769383', 'type': 'BACKGROUND', 'citation': 'Dohil R, Fidler M, Barshop BA, Gangoiti J, Deutsch R, Martin M, Schneider JA. Understanding intestinal cysteamine bitartrate absorption. J Pediatr. 2006 Jun;148(6):764-9. doi: 10.1016/j.jpeds.2006.01.050.'}, {'pmid': '17229040', 'type': 'BACKGROUND', 'citation': 'Fidler MC, Barshop BA, Gangoiti JA, Deutsch R, Martin M, Schneider JA, Dohil R. Pharmacokinetics of cysteamine bitartrate following gastrointestinal infusion. Br J Clin Pharmacol. 2007 Jan;63(1):36-40. doi: 10.1111/j.1365-2125.2006.02734.x.'}, {'pmid': '16252107', 'type': 'BACKGROUND', 'citation': 'Levtchenko EN, van Dael CM, de Graaf-Hess AC, Wilmer MJ, van den Heuvel LP, Monnens LA, Blom HJ. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006 Jan;21(1):110-3. doi: 10.1007/s00467-005-2052-0. Epub 2005 Oct 27.'}], 'seeAlsoLinks': [{'url': 'http://www.procysbi.com', 'label': 'RP103 (marketed as PROCYSBI) is now approved by the US FDA for management of nephropathic cystinosis in patients 6 years and older'}]}, 'descriptionModule': {'briefSummary': "Cystinosis is an inherited disease that if untreated, results in kidney failure as early as the first decade of life. The current marketed therapy is Cystagon® (cysteamine bitartrate) which must be taken every six hours for the rest of the patient's life to prevent complications of cystinosis. RP103 is a formulation of cysteamine bitartrate that is being studied to see if it may be able to be given less frequently, once every 12 hours, and have similar results to four times a day Cystagon®.", 'detailedDescription': 'This is a multi-center, open-label, randomized, cross-over study to determine whether steady-state, twice a day treatment with Cysteamine Bitartrate Delayed-release Capsules(RP103) results in comparable depletion of white blood cell (WBC) cystine levels compared to the existing four times a day cysteamine treatment. It will involve up to 20 clinic visits plus intermittent home use of the RP103. Most of these clinic visits occur in clusters of 3-4 consecutive days. Eligible patients will be offered enrollment into a long-term follow up study.\n\nStudy with completed results acquired from Horizon in 2024.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Male and female subjects must have nephropathic cystinosis.\n* Subjects must be on a stable dose of Cystagon® sufficient to maintain their white blood cell (WBC) cystine level at ≤ 1.0 nmol/half-cystine/mg protein.\n* Subjects must be able to swallow their typically administered Cystagon® capsule with the capsule intact.\n* Within the last 6 months, no clinically significant change in liver function \\[i.e., ALT, AST, total bilirubin\\] and renal function \\[i.e., estimated GFR\\] at Screening as determined by the Investigator.\n* Subjects with an estimated GFR (corrected for body surface area) \\> 30 mL/min/1.73m2.\n* Sexually active female subjects of childbearing potential (i.e., not surgically sterile \\[tubal ligation, hysterectomy, or bilateral oophorectomy\\] or at least 2 years naturally postmenopausal) must agree to utilize the same acceptable form of contraception from Screening through completion of the study.\n* Subjects must be willing and able to comply with the study restrictions and requirements.\n* Subjects or their or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the study.\n\nExclusion Criteria:\n\n* Subject's age \\< 6 years old or subject's weight \\< 21 kg.\n* Subjects with a known history, currently of the following conditions or other health issues that make it, in the opinion of the investigator, unsafe for them to participate: inflammatory bowel disease (if currently active) or have had prior resection of small intestine; Heart disease (e.g., myocardial infarction, heart failure, arrhythmias or poorly controlled hypertension) 90 days prior to Screening; Active bleeding disorder 90 days prior to Screening; Malignant disease within the last 2 years.\n* Patients with a hemoglobin level \\< 10 g/dL at Screening or a level that, in the opinion of the investigator, makes it unsafe for the subject to participate.\n* Subjects receiving any form of cysteamine medication through a gastric tube.\n* Subjects who are receiving maintenance dialysis or who have had a kidney transplant.\n* Subjects who are on an active kidney transplant list or who are planning to receive a kidney transplant within 3 months of Screening.\n* Subjects with known hypersensitivity to cysteamine or penicillamine.\n* Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or have a positive serum pregnancy screen.\n* Subjects who have a made a blood donation within 30 days of Screening.\n* Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol."}, 'identificationModule': {'nctId': 'NCT01000961', 'briefTitle': 'Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis', 'organization': {'class': 'INDUSTRY', 'fullName': 'Amgen'}, 'officialTitle': 'A Randomized, Crossover Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients With Nephropathic Cystinosis', 'orgStudyIdInfo': {'id': 'RP103-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RP103 Q12H', 'interventionNames': ['Drug: Cysteamine Bitartrate Delayed-release Capsules (RP103)']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Cystagon® Q6H', 'interventionNames': ['Drug: Cystagon® (Cysteamine Bitartrate)']}], 'interventions': [{'name': 'Cystagon® (Cysteamine Bitartrate)', 'type': 'DRUG', 'description': 'Run-in Period (Weeks 1, 2, 3) and Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1:\n\nEvery 6H, supplied in 150 and 50mg capsules/Duration of Treatment: 3 weeks each period used', 'armGroupLabels': ['Cystagon® Q6H']}, {'name': 'Cysteamine Bitartrate Delayed-release Capsules (RP103)', 'type': 'DRUG', 'description': 'Period 1 (Weeks 4, 5, 6) or Period 2 (Weeks 7, 8, 9); Immediate crossover to opposite treatment than taken during Period 1:\n\nEvery 12H, supplied in 75 and 25mg capsules/Duration of Treatment: 3 weeks', 'armGroupLabels': ['RP103 Q12H']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94305', 'city': 'Stanford', 'state': 'California', 'country': 'United States', 'facility': 'Stanford University Medical School', 'geoPoint': {'lat': 37.42411, 'lon': -122.16608}}, {'zip': '30322', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': "Emory Children's Center", 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '60614', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Ann & Robert H. Lurie Children's Hospital of Chicago (formerly Children's Memorial Hospital)", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'city': 'Lyon', 'country': 'France', 'facility': 'Hospices Civils de Lyon', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'city': 'Montpellier', 'country': 'France', 'facility': 'Villeneuve-Lapeyronie Hospital', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}, {'city': 'Paris', 'country': 'France', 'facility': 'Necker Hospital', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Paris', 'country': 'France', 'facility': 'Robert Debre Hospital', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'city': 'Nijmegen', 'country': 'Netherlands', 'facility': 'Radboud University Nijmegen Medical Center', 'geoPoint': {'lat': 51.8425, 'lon': 5.85278}}], 'overallOfficials': [{'name': 'MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Amgen'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Amgen', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}