Trial · NCT00999609
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
NCT00999609, Phase 3, Active not recruiting, Spark Therapeutics, Inc.; results first posted 2018-03-26
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis. Trials, record NCT00999609. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00999609
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
- NCT number
- NCT00999609
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 31 participants, actual
- Lead sponsor
- Spark Therapeutics, Inc.
- Conditions
- Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
- Interventions
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- First posted
- 2009-10-22
- Last updated
- 2025-04-23
- Results first posted
- 2018-03-26
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Multi-luminance Mobility Testing (MLMT), Bilateral | One year (change from baseline) | The MLMT measures changes in functional vision, as assessed by the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination. MLMT was assessed using both eyes at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). Each light level was assigned a score code ranging from 0 to 6. A higher score indicated that a subject was able to pass the MLMT at a lower light level. A score of -1 was assigned to those who could not pass MLMT at 400 lux. The MLMT of each subject was videotaped and assessed by independent graders. The MLMT score was determined by the lowest light level at which the subject was able to pass the MLMT. The MLMT score change was defined as the difference between the score at Baseline and the score at Year 1. A positive MLMT score change from Baseline to Year 1 visit indicated that the subject was able to complete the MLMT at a lower light level. |
| Secondary | Full-field Light Sensitivity Threshold (FST) Testing: White Light | One year (change from baseline) | Measures the light sensitivity of the entire visual field by recording the luminance at which a subject reliably reports seeing the dimmest flash. |
| Secondary | Multi-luminance Mobility Testing (Monocular) | One year (change from baseline) | The MLMT measures changes in functional vision, as assessed by the ability to navigate a course accurately and at a reasonable pace at different levels of environmental illumination. MLMT was assessed using the first eye at 1 or more of 7 levels of illumination, ranging from 400 lux (a brightly lit office) to 1 lux (a moonless summer night). Each light level was assigned a score code ranging from 0 to 6. A higher score indicated that a subject was able to pass the MLMT at a lower light level. A score of -1 was assigned to those who could not pass MLMT at 400 lux. The MLMT of each subject was videotaped and assessed by independent graders. The MLMT score was determined by the lowest light level at which the subject was able to pass the MLMT. The MLMT score change was defined as the difference between the score at Baseline and the score at Year 1. A positive MLMT score change from Baseline to Year 1 visit indicated that the subject was able to complete the MLMT at a lower light level. |
| Secondary | Visual Acuity | One year (change from baseline) | Measurement of the sharpness of vision, determined by the ability to read letters on a standardized chart from a specified distance. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Multi-luminance Mobility Testing (MLMT), Bilateral | One year (change from baseline) | Score Change in Light Levels | MEAN | |
| SECONDARY | Full-field Light Sensitivity Threshold (FST) Testing: White Light | One year (change from baseline) | log10(cd.s/(m^2)) | MEAN | |
| SECONDARY | Multi-luminance Mobility Testing (Monocular) | One year (change from baseline) | Score Change in Light Levels | MEAN | |
| SECONDARY | Visual Acuity | One year (change from baseline) | LogMAR | MEAN |
Source ClinicalTrials.gov
Results by group8
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Multi-luminance Mobility Testing (MLMT), Bilateral | Intervention | 1.8 | Standard Deviation 1.1 | Score Change in Light Levels | |
| Multi-luminance Mobility Testing (MLMT), Bilateral | Control | 0.2 | Standard Deviation 1.0 | Score Change in Light Levels | |
| Full-field Light Sensitivity Threshold (FST) Testing: White Light | Intervention | -2.08 | Standard Error 0.29 | log10(cd.s/(m^2)) | |
| Full-field Light Sensitivity Threshold (FST) Testing: White Light | Control | 0.04 | Standard Error 0.44 | log10(cd.s/(m^2)) | |
| Multi-luminance Mobility Testing (Monocular) | Intervention | 1.9 | Standard Deviation 1.2 | Score Change in Light Levels | |
| Multi-luminance Mobility Testing (Monocular) | Control | 0.2 | Standard Deviation 0.6 | Score Change in Light Levels | |
| Visual Acuity | Intervention | -0.16 | Standard Error 0.07 | LogMAR | |
| Visual Acuity | Control | 0.01 | Standard Error 0.10 | LogMAR |
Source ClinicalTrials.gov
Participant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Intervention | 21 | |
| Overall Study | STARTED | Control | 10 | |
| Overall Study | COMPLETED | Intervention | 20 | |
| Overall Study | COMPLETED | Control | 9 | |
| Overall Study | NOT COMPLETED | Intervention | 1 | |
| Overall Study | NOT COMPLETED | Control | 1 |
Source ClinicalTrials.gov
Adverse events38
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 | Cataract | Eye disorders | Intervention | 3 | 20 | 4 |
| other | Cataract | Eye disorders | Control | 0 | 9 | 0 |
| other | Elevated intraocular pressure | Eye disorders | Intervention | 4 | 20 | 5 |
| other | Elevated intraocular pressure | Eye disorders | Control | 0 | 9 | 0 |
| other | Retinal tear | Eye disorders | Intervention | 2 | 20 | 2 |
| other | Retinal tear | Eye disorders | Control | 0 | 9 | 0 |
| other | Eye inflammation | Eye disorders | Intervention | 2 | 20 | 6 |
| other | Eye inflammation | Eye disorders | Control | 0 | 9 | 0 |
| other | Conjunctival cyst | Eye disorders | Intervention | 1 | 20 | 1 |
| other | Conjunctival cyst | Eye disorders | Control | 0 | 9 | 0 |
1 to 10 of 38Source ClinicalTrials.gov
See all 38 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Intervention | 0 | 21 |
| Total, all-cause mortality | Control | 0 | 10 |
| Total, other adverse events | Intervention | 13 | 20 |
| Total, other adverse events | Control | 1 | 9 |
| Total, serious adverse events | Intervention | 2 | 20 |
| Total, serious adverse events | Control | 0 | 9 |
Source ClinicalTrials.gov
Papers10
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Intervention vs Control: deaths from any cause | Trial comparisons |
| Intervention vs Control: serious adverse events | Trial comparisons |
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/NCT00999609
GET https://conductscience.org/api/trials/v1/NCT00999609/events?page=2