Single Ascending Dose Study in Participants With LCA10

• Status: Recruiting
• Trial end date: 2024-03-22
• Target enrollment: 0
• Participant gender: All

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").

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Allergan; Editas Medicine, Inc.
• Collaborator: Editas Medicine, Inc.

Criteria

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.

• Best corrected visual acuity of light perception to 0.4 logMAR (20/50 Snellen equivalent).

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