Single Ascending Dose Study in Participants With LCA10

Editas Medicine

Status and phase

Active, not recruiting

Phase 2

Phase 1

Conditions

Eye Diseases, Hereditary

Inherited Retinal Dystrophies

Eye Disorders Congenital

Retinal Degeneration

Leber Congenital Amaurosis 10

Vision Disorders

Retinal Disease

Treatments

Drug: EDIT-101

Study type

Interventional

Funder types

Industry

Identifiers

NCT03872479

1991-201-008

Details and patient eligibility

About

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").

Full description

This is an open-label, single ascending dose study of EDIT-101 in adult and pediatric (ie, ages 3 to 17) participants with LCA10-IVS26. Up to 34 participants will be enrolled in up to 5 cohorts to evaluate up to 3 dose levels of EDIT-101 in this study. EDIT-101 is a novel gene editing product designed to eliminate the mutation on the CEP290 gene that results in the retinal degeneration that defines LCA10-IVS26.

Enrollment

34 estimated patients

Sex

All

Ages

3+ years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

34 participants in 5 patient groups

Adults Low Dose

Experimental group

Description: