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NCT00001213 · ClinicalTrials.gov registry record · Phase 2
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
A Phase 2 study, sponsored by National Eye Institute (NEI).
- Completed
- Registry status
- Phase 2
- Development phase
- 328
- Enrollment target
NCT00001213: Completed Phase 2 study, sponsored by National Eye Institute (NEI).
NCT00001213 is a Phase 2 study that has completed, run by National Eye Institute (NEI). The registered enrollment target is 328 participants, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (147% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001213, a Phase 2 study, has completed, sponsored by National Eye Institute (NEI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 328 participants
- Enrollment target
Study Summary
Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the eye over the pupil and iris. After 10 to 20 years, the corneas of some patients become so packed with crystals that the surfaces may become irregular, occasionally causing small, painful breaks. Patients enrolled in a NIH study on cystinosis are receiving the drug cysteamine. Taken by mouth, this drug reduces cystine in some tissues, but not in the cornea. This study began in 1986 to test whether cysteamine eye drops could prevent or reduce corneal cystine crystals in these patients. The drops have been very effective in removing crystals and reducing pain in patients who take the medication as directed. Patients who do not take the medication as prescribed do not benefit. After the effectiveness of the drops was proven, the main purpose was modified to continue to evaluate the long-term safety and effectiveness of cysteamine eye drops for treating cystine crystals in the corneas of patients with cystinosis until the drops are approved by the Food and Drug Administration (FDA). When the New Drug Application (NDA) for the Sigma-Tau standard formulation is granted, this protocol will be terminated.
Primary Outcome
Since efficacy of ophthalmic cysteamine was established and a New Drug Application (NDA) filed, the post-hoc primary outcome measure is the evaluation of safety information. There was no specified time frame for this outcome measure, as safety data was being collected until the drug became available for commercial purchase in May 2013.
Interventions
- DRUG Cysteamine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 328 participants |
| Start Date | 1986-04 |
| Est. Completion | 2013-07 |
| Phase | Phase 2 |
What the finished NCT00001213 record still lists
NCT00001213 is an interventional study that assigns participants to a tested intervention. The registered 328 participants enrollment target is mid-sized for trials with a published cap, above the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (147% higher).
The record links to 0 conditions, and to 1 intervention - of which Cysteamine is the first listed.
NCT00001213 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00001213 about?
NCT00001213 is a clinical study titled "Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis". Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body tissues. This chemical also accumulates in the cornea-the covering of the...
What is the current status of trial NCT00001213?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 328 participants. The study started on 1986-04. Estimated completion is 2013-07.
What interventions are being tested in trial NCT00001213?
The interventions under investigation include: Cysteamine (DRUG).
Who is sponsoring clinical trial NCT00001213?
This trial is sponsored by National Eye Institute (NEI), which has 221 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00001213's enrollment target sits among peer trials
328 146th of 2000 higher than 1,855 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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