Single Ascending Dose Study in Participants With LCA10

Status unconfirmed · Phase 1/Phase 2

Conditions studied: Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital

In brief

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").

Key facts

Study ID

NCT03872479

Run by

Editas Medicine, Inc.

People needed

34

Starts

2019-09-26

Expected to finish

2025-05-23

Last updated by the study team

2022-12-05

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Bascom Palmer Eye Institute — Miami, Florida, United States
• Massachusetts Eye and Ear Infirmary — Boston, Massachusetts, United States
• W.K. Kellogg Eye Center - University of Michigan — Ann Arbor, Michigan, United States
• Casey Eye Institute - OSHU — Portland, Oregon, United States
• University of Pennsylvania — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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