Gene Therapy for Leber Congenital Amaurosis

Recruiting at 4 trial locations

Overseen ByEditas Medicine's Clinical Trial Team

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Waitlist Available

Sponsor: Editas Medicine, Inc.

No Placebo Group

Trial Summary

What is the purpose of this trial?

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").

Eligibility Criteria

This trial is for males and females aged 3 or older with a specific genetic form of retinal degeneration (LCA10) due to mutations in the CEP290 gene. Participants must have severe vision loss, but not other disease-causing mutations or conditions that could affect the study's outcome like recent vaccinations, unwillingness to take oral prednisone, or prior similar treatments.

Inclusion Criteria

I am 3 years or older with a specific genetic eye condition.

The participant has very poor vision in the study eye, with a measurement of 20/800 or worse.

Participants, other than those specifically being studied, must have a visual acuity between 1.0 and 3.0 logMAR in the eye being studied.

Exclusion Criteria

I have not had any vaccinations in the last 28 days.

I passed the hardest level of a mobility test.

I have never received gene therapy or oligonucleotide treatment.

See 4 more

Treatment Details

Interventions

EDIT-101 (Gene Therapy)

Trial OverviewThe trial tests EDIT-101 delivered through an injection into the retina for safety and effectiveness. It targets patients with LCA10 caused by certain CEP290 gene mutations. The study involves giving participants one dose of EDIT-101 and observing its effects on their condition.

Participant Groups

5Treatment groups

Experimental Treatment

Group I: Pediatric Middle DoseExperimental Treatment1 Intervention