Single Ascending Dose Study in Participants With LCA10
Public ClinicalTrials.gov record NCT03872479. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26")
Study identification
- NCT ID
- NCT03872479
- Recruitment status
- Not listed
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Lead sponsor
- Editas Medicine, Inc.
- Industry
- Enrollment
- 34 participants
Conditions and interventions
Conditions
Interventions
- EDIT-101 Drug
Drug
Eligibility (public fields only)
- Age range
- 3 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 25, 2019
- Primary completion
- May 22, 2025
- Completion
- May 22, 2025
- Last update posted
- Dec 4, 2022
2019 – 2025
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Bascom Palmer Eye Institute | Miami | Florida | 33136 | — |
| Massachusetts Eye and Ear Infirmary | Boston | Massachusetts | 02114 | — |
| W.K. Kellogg Eye Center - University of Michigan | Ann Arbor | Michigan | 48105 | — |
| Casey Eye Institute - OSHU | Portland | Oregon | 97239 | — |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT03872479, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 4, 2022 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT03872479 live on ClinicalTrials.gov.