Trial · NCT05203939
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
NCT05203939, Phase 1, Phase 2, Active not recruiting, Ocugen
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis. Trials, record NCT05203939. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05203939
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
- NCT number
- NCT05203939
- Status
- Active not recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 22 participants, actual
- Lead sponsor
- Ocugen
- Conditions
- Retinitis Pigmentosa, Leber Congenital Amaurosis
- Interventions
- OCU400 Low Dose, OCU400 Med Dose, OCU400 High Dose, OCU400 Second Eye Dosing
- First posted
- 2022-01-24
- Last updated
- 2026-07-31
Source ClinicalTrials.gov
Registered outcomes19
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Study Drug-related adverse events (SDAE) | 1 year | Counts, frequencies and percentages of SDAEs. SDAE is a primary adverse event of interest and defined as AEs and SAEs that are direct subjects to the Study Drug only. |
| Primary | Treatment-Emergent adverse events (TEAEs) | 1 year | Counts, frequencies and percentages TEAEs. TEAEs are defined as an event that was not present prior to administration of the dose of study drug and present after the dose, or if it represents the exacerbation of an event that was present prior to the dose. |
| Primary | Serious adverse events (SAEs) | 1 year | Counts, frequencies and percentages of SAEs including Resulted in Death, Life-threatening, Hospitalization, Disabling/incapacitating, Congenital anomaly or birth defect and medically significant AEs ( AE that did not meet any of the above criteria but could have jeopardized the subject and might have required medical or surgical intervention to prevent one of the outcomes listed above). |
| Secondary | Best-corrected visual acuity (BCVA) | 1 year (Changes from baseline) | Measured as the ETDRS letter score on the EVA tester or E-ETDRS charts. Electronic ETDRS Visual Acuity Testing Protocol will be followed (confidential). |
| Secondary | Low-luminance visual acuity (LLVA) | 1 year (Changes from baseline) | Electronic Visual Acuity Tester (EVA) and a Sponsor specific Low-Luminance lens will be used. Early Treatment of Diabetic Retinopathy Study (ETDRS) will also be accepted as a backup. |
| Secondary | Slit-lamp biomicroscopy | 1 year (Changes from baseline) | Changes in visual function. |
| Secondary | Intraocular pressure (IOP) | 1 year (Changes from baseline) | IOP measurement by applanation or rebound tonometry. Confirmation with Goldmann tonometer if IOP reading is outside the normal range (8-21mmHg). |
| Secondary | Indirect ophthalmoscopy | 1 year (Changes from baseline) | If visual acuity is so poor that the participant is unable to count fingers or perceive hand motion, light perception will be tested with the indirect ophthalmoscope as the light source. |
| Secondary | anti-AAV5 (anti Adeno-associated virus type 5) | 1 year | Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory. |
| Secondary | anti-hNR2E3 antibodies (hNR2E3 gene) | 1 year | Blood samples will be collected for the assessment. These samples will be analyzed using validated assays at a bioanalytical laboratory. |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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/NCT05203939
GET https://conductscience.org/api/trials/v1/NCT05203939/registered