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UnknownNCT03872479

Single Ascending Dose Study in Participants With LCA10

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")

Status
Unknown
Phase
Phase 1 / Phase 2
Study type
Interventional
Enrollment
34 (estimated)
Sponsor
Editas Medicine, Inc. · Industry
Sex
All
Age
3 Years
Healthy volunteers
Not accepted

Summary

The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").

Detailed description

This is an open-label, single ascending dose study of EDIT-101 in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Up to 34 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of EDIT-101 in this study. EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.

Conditions

Interventions

TypeNameDescription
DRUGEDIT-101Participants will receive a single dose of EDIT-101 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study.

Timeline

Start date
2019-09-26
Primary completion
2025-05-23
Completion
2025-05-23
First posted
2019-03-13
Last updated
2022-12-05

Locations

5 sites across 1 country: United States

Regulatory

Source: ClinicalTrials.gov record NCT03872479. Inclusion in this directory is not an endorsement.