Status:
UNKNOWN
Single Ascending Dose Study in Participants With LCA10
Lead Sponsor:
Editas Medicine, Inc.
Conditions:
Leber Congenital Amaurosis 10
Inherited Retinal Dystrophies
Eligibility:
All Genders
3+ years
Phase:
PHASE1
PHASE2
Brief 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 hom...
Detailed 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 eva...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
September 26 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 23 2025
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT03872479
Start Date
September 26 2019
End Date
May 23 2025
Last Update
December 5 2022
Active Locations (5)
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1
Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
2
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114
3
W.K. Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, United States, 48105
4
Casey Eye Institute - OSHU
Portland, Oregon, United States, 97239