Cystinosis Clinical Trials

42 clinical trials for Cystinosis across international registries, including 9 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Cystinosis and Mitochondrial Metabolism — RECRUITING · NA
• Use of Cysteamine in the Treatment of Cystinosis — RECRUITING
• European Cystinosis Cohort — RECRUITING
• Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford — RECRUITING
• Development of Health-related Quality of Life Instrument for Patients With Cystinosis — RECRUITING
• A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04 — RECRUITING
• National Registry of Rare Kidney Diseases — RECRUITING
• Early Trial of Allogeneic Hematopoietic Stem Cell Transplantation for Patients Who Will Receive a Kidney Transplant From the Same Donor — RECRUITING · PHASE1
• A PHASE 2, SINGLE CENTER, OPEN-LABEL, MULTIPLE DOSE ESCALATION STUDY TO EVALUATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF DAILY SUBCUTANEOUSLY ADMINISTERED ELX-02 IN PATIENTS WITH NEPHROPATHIC CYSTINOSIS BEARING ONE OR MORE CTNS GENE (CYSTINOSIN) NONSENSE MUTATIONS — RECRUITING · PHASE2
• 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 — COMPLETED · PHASE3
• Stem Cell Gene Therapy for Cystinosis — COMPLETED · PHASE1
• European Cystinosis Cohort 2 — NOT_YET_RECRUITING
• Early Check: Expanded Screening in Newborns — ACTIVE_NOT_RECRUITING
• PK and PD Study of NPI-001 and Cysteamine Bitartrate — ACTIVE_NOT_RECRUITING · PHASE1
• Gastrointestinal involvement and diet in cystinosis — NOT_YET_RECRUITING
• Clinical and Biological Study of Sub-pigmentation During Infantile Cystinosis — COMPLETED · NA
• A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications — COMPLETED · NA
• Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients — COMPLETED
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) — COMPLETED · PHASE3
• Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis — COMPLETED · PHASE3
• Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis — COMPLETED · PHASE3
• Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis — COMPLETED · PHASE1
• Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis — COMPLETED · PHASE3
• The Effect of Exercise on Muscle Dysfunction in Cystinosis — COMPLETED · NA
• Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2) — COMPLETED · PHASE3
• Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy — COMPLETED · NA
• Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria — OTHER · NA
• A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis — TERMINATED · PHASE2
• Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis — WITHDRAWN · PHASE1
• Kidney Transplantation in Patients With Cystinosis — COMPLETED
• The Role of Susceptibility to Thrombosis in the Pseudotumor Cerebri of Nephropathic Cystinosis: A Case-Control Study — COMPLETED
• Salivary Proteins in Disease and Health — COMPLETED
• The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients — COMPLETED · PHASE1
• Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis — COMPLETED · NA
• Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis — COMPLETED · PHASE2
• Cure Cystinosis International Registry — OTHER
• Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ — COMPLETED · NA
• N Acetyl Cysteine for Cystinosis Patients — COMPLETED · PHASE4
• New Cysteamine Eye Drops Formulation to Treat Corneal Crystals in Cystinosis — COMPLETED · PHASE1
• Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis — COMPLETED
• Evaluation of Fanconi Syndrome and Cystinosis — COMPLETED

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