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Single Ascending Dose Study in Participants With LCA10

NCT03872479 · Status: UNKNOWN · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 34

Last updated 2022-12-05

No results posted yet for this study

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").

Conditions

  • Leber Congenital Amaurosis 10
  • Inherited Retinal Dystrophies
  • Eye Diseases, Hereditary
  • Retinal Disease
  • Retinal Degeneration
  • Vision Disorders
  • Eye Disorders Congenital

Interventions

DRUG

EDIT-101

Participants 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.

Sponsors & Collaborators

  • Editas Medicine, Inc.

    lead INDUSTRY

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
3 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-09-26
Primary Completion
2025-05-23
Completion
2025-05-23
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03872479 on ClinicalTrials.gov