Trial · NCT04855045
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
NCT04855045, Phase 2, Phase 3, Unknown, ProQR Therapeutics
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. Trials, record NCT04855045. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04855045
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
- NCT number
- NCT04855045
- Status
- Unknown
- Phases
- Phase 2, Phase 3
- Enrollment
- 15 participants, estimated
- Lead sponsor
- ProQR Therapeutics
- Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
- Interventions
- sepofarsen
- First posted
- 2021-04-22
- Last updated
- 2022-03-25
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence and severity of ocular adverse events (AEs) | 24 months | Incidence and severity of ocular adverse events (AEs) |
| Primary | Incidence and severity of non-ocular adverse events (AEs) | 24 months | Incidence and severity of non-ocular adverse events (AEs) |
| Secondary | Change from baseline to Month 12 in Best-corrected visual acuity (BCVA) | 12 months | Mean change in BCVA relative to baseline after 12 months of treatment |
| Secondary | Change from baseline to Month 12 in retinal sensitivity measured by Full-field stimulus testing (FST) | 12 months | Mean change in retinal sensitivity measured by FST relative to baseline after 12 months of treatment |
Source ClinicalTrials.gov
Outcomes0
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/NCT04855045
GET https://conductscience.org/api/trials/v1/NCT04855045/registered