Gene Therapy for Leber Congenital Amaurosis

Not currently recruiting at 4 trial locations

Overseen ByEditas Medicine's Clinical Trial Team

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Editas Medicine, Inc.

Must not be taking: Corticosteroids

Disqualifiers: Other mutations, Infections, Steroid response, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy called EDIT-101 to determine its safety and effectiveness for individuals with Leber Congenital Amaurosis 10 (LCA10), a rare eye condition. Specific changes in the CEP290 gene cause this condition, leading to severe vision loss. Participants will receive a single dose of the treatment directly into the eye. The trial seeks individuals aged 3 and older with severe vision problems due to LCA10 and a specific genetic mutation. As a Phase 1/Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. However, it mentions that you must be willing to take oral prednisone, which might interact with other medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that EDIT-101 is likely to be safe for humans?

Research has shown that EDIT-101, the treatment under study, was safe in earlier studies, with most patients not experiencing serious side effects. These studies also found some signs of potential vision improvement, which is promising. Although the treatment is still being tested, these early results suggest it is generally safe for people.¹ ² ³ ⁴ ⁵

Why do researchers think this study treatment might be promising for LCA?

Unlike standard treatments for Leber Congenital Amaurosis, which often involve vision aids or vitamin supplements, EDIT-101 is a groundbreaking gene therapy targeting the root cause of the disease. This treatment works by using CRISPR technology to directly edit the genetic mutations responsible for the condition, offering the potential for a more permanent solution. Researchers are excited because this approach could restore vision by correcting the genetic defect itself, rather than just managing symptoms. Additionally, the delivery method via subretinal injection ensures precise targeting of the affected cells, which is a significant advancement in treatment precision.

What evidence suggests that EDIT-101 could be an effective treatment for LCA10?

Research has shown that EDIT-101 holds promise for treating Leber congenital amaurosis 10 (LCA10), a serious eye disorder. In early animal tests, EDIT-101 achieved the necessary level of gene editing to improve vision. Initial results from human trials suggest that patients treated with EDIT-101 experienced consistent improvements in eyesight clarity, as measured by best-corrected visual acuity (BCVA). Furthermore, the treatment has proven safe at different dose levels, which are being tested in this trial across various arms, including Adults High Dose and Pediatric Middle Dose. These findings support the potential effectiveness of EDIT-101 for individuals with LCA10.¹ ² ⁴ ⁶ ⁷

Are You a Good Fit for This Trial?

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.

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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single ascending dose of EDIT-101 administered via subretinal injection

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Multiple visits (in-person and virtual)

What Are the Treatments Tested in This Trial?

Interventions

EDIT-101

Trial Overview

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.