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Gene Therapy

Gene Therapy for Leber Congenital Amaurosis

Phase 1 & 2

Waitlist Available

Research Sponsored by Editas Medicine, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

Achieves a passing score for the mobility course at the most difficult level

Prior gene therapy or oligonucleotide treatment

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new gene therapy for people with a specific genetic mutation that causes Leber Congenital Amaurosis, a severe form of vision loss.

Who is the study for?

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.

What is being tested?

The 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.

What are the potential side effects?

Potential side effects are not explicitly listed but may include typical risks associated with subretinal injections such as eye inflammation, infection risk increase following corticosteroid exposure, and possible adverse reactions related to the EDIT-101 treatment.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I passed the hardest level of a mobility test.

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I have never received gene therapy or oligonucleotide treatment.

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I have other genetic mutations known to cause disease.

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I currently have an eye or systemic infection or inflammation.

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I cannot or do not want to take oral prednisone.

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My eye pressure increased above 25 mm Hg after using steroids.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Change from baseline in LogMAR measurement of BCVA

Change from baseline in Mobility course score

Change from baseline in Patient Global Impressions of Change score

+7 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.