Cystinosis
39
7
9
24
Key Insights
Highlights
Success Rate
96% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
2.6%
1 terminated out of 39 trials
96.0%
+9.5% vs benchmark
15%
6 trials in Phase 3/4
29%
7 of 24 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 24 completed trials
Clinical Trials (39)
Use of Cysteamine in the Treatment of Cystinosis
European Cystinosis Cohort
European Cystinosis Cohort 2
Early Check: Expanded Screening in Newborns
PK and PD Study of NPI-001 and Cysteamine Bitartrate
Clinical and Biological Study of Sub-pigmentation During Infantile Cystinosis
Stem Cell Gene Therapy for Cystinosis
Cystinosis and Mitochondrial Metabolism
A Cohort of Patients With Cystinosis : Compliance to Cysteamine and Neurological Complications
Neuromuscular Characterisation in Late Adolescent and Adult Cystinosis Patients
Baby Detect : Genomic Newborn Screening
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis
Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103)
Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis
Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis
Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis
The Effect of Exercise on Muscle Dysfunction in Cystinosis
Development of Health-related Quality of Life Instrument for Patients With Cystinosis
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04