UnknownPhase 1/2

Single Ascending Dose Study in Participants With LCA10

United States34 participantsStarted 2019-09-26

Plain-language 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").

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Male or female * At least 3 years of age at screening with CEP290-related retinal degeneration caused by a homozygous or compound heterozygous mutation involving c.2991+1655A\>G in IVS26 of the CEP290 gene. * Visual Acuity: * Sentinel participant will have severe vision loss with a logMAR BCVA of ≥1.6 to 3.9 (20/800 or worse to LP) in the study eye * Non-sentinel participants must have BCVA between 1.0 - 3.0 logMAR in the study eye Exclusion Criteria: * Other known disease-causing mutations * Achieves a passing score for the mobility course at the most difficult level * In either eye, active systemic or ocular/intraocular infection or inflammation * In either eye, history of steroid-responsive intraocular pressure with increases \> 25 mm Hg following corticosteroid exposure * Any vaccination/immunization in the last 28 days before screening * Inability or unwillingness to take oral prednisone * Prior gene therapy or oligonucleotide treatment

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Frequency of Adverse Events related to EDIT-101

Timeframe: 1 year

Number of participants experiencing procedural related adverse events

Timeframe: 1 year

Incidence of dose limiting toxicities

Timeframe: 1 year

Trial details

NCT IDNCT03872479

SponsorEditas Medicine, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-05-23

View on ClinicalTrials.gov More Leber Congenital Amaurosis 10 trials

Plain-language summary

Who can participate

Age range

3 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.