Not applicableUnknownNCT01327807What this trial is testingCure Cystinosis International RegistryWho this might be right forCystinosisNephropathic CystinosisRenal Fanconi Syndrome Cystinosis Research Foundation 750
Not applicableStudy completedNCT02766855What this trial is testingEfficacy of Topical Cysteamine in Nephropathic CyctinosisWho this might be right forNephropathic CyctinosisCorneal Cystine Crystals The Eye Center and The Eye Foundation for Research in Ophthalmology 26
Testing effectiveness (Phase 2)Looking for participantsNCT06910813What this trial is testingDFT383 in Pediatric Participants With Nephropathic CystinosisWho this might be right forNephropathic Cystinosis Novartis Pharmaceuticals 30
Not applicableStudy completedNCT06027385What this trial is testingGenetic Newborn Screening for Cystinosis and Spinal Muscular AtrophyWho this might be right forCystinosisCystinosis, NephropathicSpinal Muscular Atrophy Cystinose Stiftung 300,000
Testing effectiveness (Phase 2)Ended earlyNCT04069260What this trial is testingELX-02 in Patients With Nephropathic CystinosisWho this might be right forGenetic DiseaseNonsense MutationCystinosis Eloxx Pharmaceuticals, Inc. 3
Not applicableStudy completedNCT00004312What this trial is testingEstablishment of a Database for Long-Term Monitoring of Patients With Nephropathic CystinosisWho this might be right forCystinosis National Center for Research Resources (NCRR) 400
Testing effectiveness (Phase 2)Study completedNCT00001213What this trial is testingCysteamine Eye Drops to Treat Corneal Crystals in CystinosisWho this might be right forCystinosis National Eye Institute (NEI) 328
Large-scale testing (Phase 3)Study completedNCT01000961What this trial is testingPhase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With CystinosisWho this might be right forCystinosis Amgen 43
Not applicableStudy completedNCT01432561What this trial is testingStudy in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™Who this might be right forCystinosisNephropathic Cystinosis University of California, San Diego 8
Large-scale testing (Phase 3)Study completedNCT01197378What this trial is testingLong-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)Who this might be right forCystinosis Amgen 60
Not applicableStudy completedNCT00071903What this trial is testingThe Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control StudyWho this might be right forPseudotumor CerebriCystinosis Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 18
Not applicableLooking for participantsNCT01793168What this trial is testingRare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordWho this might be right forRare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more Sanford Health 20,000
Testing effectiveness (Phase 2)Study completedNCT00872729What this trial is testingPilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With CystinosisWho this might be right forCystinosis Amgen 9
Large-scale testing (Phase 3)Study completedNCT01733316What this trial is testingOpen-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in CystinosisWho this might be right forCystinosis Amgen 41
Not applicableEnrolling By InvitationNCT04246060What this trial is testingObservational Study to Assess the Quality of Life in Nephropathic Cystinosis PatientsWho this might be right forNephropathic Cystinosis Chiesi SA/NV 31
Not applicableLooking for participantsNCT03919981What this trial is testingCYSTEA-BONE Clinical StudyWho this might be right forNephropathic Cystinosis Hospices Civils de Lyon 50
Large-scale testing (Phase 3)Study completedNCT01744782What this trial is testingSafety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With CystinosisWho this might be right forCystinosis Amgen 17
Not applicableStudy completedNCT00074516What this trial is testingKidney Transplantation in Patients With CystinosisWho this might be right forCystinosis National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) 11