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Home › Conditions › Cystinosis

Cystinosis

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for Cystinosis

7 trials tracked for Cystinosis: 4 in phase 3 or 4 and 1 with published results. The most-cited published study has 54 citations.

7Trials tracked
4Phase 3 & 4
0Recruiting
1With published results
Phase distribution
Phase 3 4 Phase 2 2 Other / NA 1
  1. Phase 3 Long-Term Safety Follow-up Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) Completed · 54 cited
  2. Phase 3 Open-Label, Safety and Superior Effectiveness Study of Cysteamine Bitartrate Delayed-Release Capsules (RP103) in Cystinosis Completed
  3. Phase 3 Safety/Effectiveness Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Cysteamine Treatment Naive Patients With Cystinosis Completed
  4. Phase 3 Phase 3 Study of Cysteamine Bitartrate Delayed-release (RP103) Compared to Cystagon® in Patients With Cystinosis Completed
  5. Phase 2 Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis Completed
  6. Phase 2 Pilot Study of Safety, Tolerability, Pharmacokinetics/Pharmacodynamics of RP103 Compared to Cystagon® in Patients With Cystinosis Completed
Show 1 more trials
  1. N/A Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™ Completed

Showing the 7 most-cited and recently-updated of 7 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.

What the trials found For clinicians

Cystinosis: what the trials found

Cysteamine Bitartrate (Cystagon®) is an established intervention for cystinosis, with clinical data supporting its use in managing the condition [1, 4].

RP103 has been evaluated as a treatment option. In studies comparing RP103 to Cystagon®, RP103 demonstrated significantly lower steady-state white blood cell (WBC) cystine levels compared to Cystagon® (p=0.0001) 4.

Data regarding RP103 also indicates a statistically significant reduction in the average difference between morning and non-morning log WBC cystine values (-0.229; 0.080, p=0.0048) 3.

Recent results — preliminary, needs further review

  • Cysteamine was evaluated for corneal cystine crystal score (CCCS) response in a study of 328 patients, where 98 eyes showed a response 6.
  • Cysteamine bitartrate was also investigated in a small cohort (N=8) 7.

For the clinician treating this condition

  • Both Cysteamine Bitartrate and RP103 are established treatments for cystinosis.
  • RP103 demonstrated significantly lower steady-state white blood cell cystine levels compared to Cystagon® (p=0.0001) 4.
  • RP103 showed a statistically significant reduction in the difference between morning and non-morning log WBC cystine values 3.

AI synthesis of 4 cited trials, updated Jun 29, 2026. Informational only — not medical advice; trial data sourced from ClinicalTrials.gov. How we use AI.

HCP Mode — summaries include clinical detail, trial data, and statistical outcomes.
Patient Mode — summaries use plain language, avoiding clinical jargon.
Cystinosis patients show larger neural conflict monitoring differences versus controls in observational study
Nephrology
Cystinosis patients show larger neural conflict monitoring differences versus controls in observational study Study finds differences in brain conflict monitoring in people with cystinosis
An observational study of 36 individuals with cystinosis and 39 age-matched controls found that those with cystinosis presented larger diffe…
People with cystinosis show slower, less accurate brain responses to conflicting information compared to peers, requiring more mental effort…
Frontiers Apr 1, 2026