Trial · NCT00001213
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
NCT00001213, Phase 2, Completed, National Eye Institute (NEI); results first posted 2014-07-22
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis. Trials, record NCT00001213. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00001213
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
- NCT number
- NCT00001213
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 328 participants, actual
- Lead sponsor
- National Eye Institute (NEI)
- Conditions
- Cystinosis
- Interventions
- Cysteamine
- First posted
- 1999-11-04
- Last updated
- 2014-07-22
- Results first posted
- 2014-07-22
- Limitations and caveats
Due to the progressive nature of the drug's development, this protocol reflects the culmination of cysteamine studies performed at NIH. The combined analyses of the data is presented and not the data or analyses of one individual protocol.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Serious and Non-Serious Adverse Events | Any Time Point up to 27 Years | 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. |
| Primary | Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response | Any Time Point Up to 19 Years | Response is defined as a decrease from baseline of at least 1 in Corneal Cystine Crystal Score (CCCS) at any time on study when baseline CCCS is greater than or equal to 1, or CCCS does not increase at least 1 at any time on study when baseline CCCS is less than 1. The CCCS is based on a library of slit-lamp photographs of corneas with increasing crystal densities (0-3). Slit-lamp photos were to be taken to assess the extent of the corneal crystal accumulation. To minimize bias when assessing the extent of corneal crystal accumulation, photos were centrally graded at the National Eye Institute (NEI) where each photo was graded independently by masked graders. If more than one CCCS was recorded in a given study year, the highest (worst) CCCS value was used for that year. The results were obtained from a combined analyses of the NIH cysteamine studies evaluating various cysteamine ophthalmic solution formulations from 1986 through 2005. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Serious and Non-Serious Adverse Events | Any Time Point up to 27 Years | participants | NUMBER | |
| PRIMARY | Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response | Any Time Point Up to 19 Years | One hundred sixty-one (161) participants were analyzed in the pre-specified intent-to-treat population \[defined as patients who received study medication (between 1986 and 2005), and had a baseline and a post-baseline CCCS value\]. After 2005, all participants enrolled received open-label treatment and only safety data was obtained. | eyes | NUMBER |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Serious and Non-Serious Adverse Events | Cysteamine Topical Solution | 257 | participants | ||
| Number of Eyes With a Corneal Cystine Crystal Score (CCCS) Response | Cysteamine Topical Solution | 98 | eyes |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cysteamine Topical Solution | 328 | |
| Overall Study | COMPLETED | Cysteamine Topical Solution | 310 | |
| Overall Study | NOT COMPLETED | Cysteamine Topical Solution | 18 |
Source ClinicalTrials.gov
Adverse events48
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Photophobia | Eye disorders | Cysteamine Topical Solution | 239 | 328 | 239 |
| other | Conjunctival hyperaemia | Eye disorders | Cysteamine Topical Solution | 69 | 328 | 69 |
| other | Eye pain | Eye disorders | Cysteamine Topical Solution | 66 | 328 | 66 |
| other | Ocular hyperaemia | Eye disorders | Cysteamine Topical Solution | 52 | 328 | 52 |
| other | Eye irritation | Eye disorders | Cysteamine Topical Solution | 49 | 328 | 49 |
| other | Lacrimation increased | Eye disorders | Cysteamine Topical Solution | 23 | 328 | 23 |
| other | Optic disc disorder | Eye disorders | Cysteamine Topical Solution | 19 | 328 | 19 |
| other | Vision blurred | Eye disorders | Cysteamine Topical Solution | 20 | 328 | 20 |
| other | Keratitis | Eye disorders | Cysteamine Topical Solution | 19 | 328 | 19 |
| other | Dry eye | Eye disorders | Cysteamine Topical Solution | 14 | 328 | 14 |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cysteamine Topical Solution | ||
| Total, other adverse events | Cysteamine Topical Solution | 257 | 328 |
| Total, serious adverse events | Cysteamine Topical Solution | 11 | 328 |
Source ClinicalTrials.gov
Papers5
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00001213
GET https://conductscience.org/api/trials/v1/NCT00001213/events?page=2