Cystinosis
Pipeline by Development Stage
Competitive Landscape
8 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (12)
Total enrollment: 997 patients across 12 trials
Cystadrops in Pediatric Cystinosis Patients From Six Months to Less Than Two Years Old (SCOB2)
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Randomized Study of New Formulation Ophthalmic Cysteamine Hydrochloride for Corneal Cystine Accumulation in Patients With Cystinosis
Establishment of a Database for Long-Term Monitoring of Patients With Nephropathic Cystinosis
Use of Cysteamine in the Treatment of Cystinosis
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Key Insights
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.