Single Ascending Dose Study in Participants With LCA10
Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of EDIT-101 in Adult and Pediatric Participants With Leber Congenital Amaurosis Type 10 (LCA10), With Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in Intron 26 (IVS26) of the CEP290 Gene ("LCA10-IVS26")
1 other identifier
interventional
34
1 country
5
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 homozygous or compound heterozygous mutation involving c.2991+1655A\>G in intron 26 of the CEP290 gene ("LCA10-IVS26").
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Sep 2019
Longer than P75 for phase_1
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 12, 2018
CompletedFirst Posted
Study publicly available on registry
March 13, 2019
CompletedStudy Start
First participant enrolled
September 26, 2019
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 23, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
May 23, 2025
CompletedDecember 5, 2022
December 1, 2022
5.7 years
October 12, 2018
December 2, 2022
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Frequency of Adverse Events related to EDIT-101
1 year
Number of participants experiencing procedural related adverse events
1 year
Incidence of dose limiting toxicities
1 year
Secondary Outcomes (16)
Maximum tolerated dose as determined by occurrence of dose limiting toxicities
1 year
Change from baseline in Mobility course score
1 year
Change from baseline in LogMAR measurement of BCVA
1 year
Change from baseline in pupillary response
1 year
Change from baseline in dark adapted visual sensitivity using Full field light sensitivity threshold (FST)
1 year
- +11 more secondary outcomes
Study Arms (5)
Adults Low Dose
EXPERIMENTALSingle dose of EDIT-101 administered by subretinal injection surgery
Adults Middle Dose
EXPERIMENTALSingle dose of EDIT-101 administered by subretinal injection surgery
Adults High Dose
EXPERIMENTALSingle dose of EDIT-101 administered by subretinal injection surgery
Pediatric Middle Dose
EXPERIMENTALSingle dose of EDIT-101 administered by subretinal injection surgery
Pediatric High Dose
EXPERIMENTALSingle dose of EDIT-101 administered by subretinal injection surgery
Interventions
Participants will receive a single dose of EDIT-101 administered via subretinal injection in the study eye. Up to 5 cohorts across 3 doses will be enrolled in this study.
Eligibility Criteria
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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Bascom Palmer Eye Institute
Miami, Florida, 33136, United States
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
W.K. Kellogg Eye Center - University of Michigan
Ann Arbor, Michigan, 48105, United States
Casey Eye Institute - OSHU
Portland, Oregon, 97239, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Related Publications (3)
Pierce EA, Aleman TS, Jayasundera KT, Ashimatey BS, Kim K, Rashid A, Jaskolka MC, Myers RL, Lam BL, Bailey ST, Comander JI, Lauer AK, Maguire AM, Pennesi ME. Gene Editing for CEP290-Associated Retinal Degeneration. N Engl J Med. 2024 Jun 6;390(21):1972-1984. doi: 10.1056/NEJMoa2309915. Epub 2024 May 6.
PMID: 38709228DERIVEDHarvey JP, Sladen PE, Yu-Wai-Man P, Cheetham ME. Induced Pluripotent Stem Cells for Inherited Optic Neuropathies-Disease Modeling and Therapeutic Development. J Neuroophthalmol. 2022 Mar 1;42(1):35-44. doi: 10.1097/WNO.0000000000001375. Epub 2021 Sep 30.
PMID: 34629400DERIVEDZhang X, Zhang D, Thompson JA, Chen SC, Huang Z, Jennings L, McLaren TL, Lamey TM, De Roach JN, Chen FK, McLenachan S. Gene correction of the CLN3 c.175G>A variant in patient-derived induced pluripotent stem cells prevents pathological changes in retinal organoids. Mol Genet Genomic Med. 2021 Mar;9(3):e1601. doi: 10.1002/mgg3.1601. Epub 2021 Jan 26.
PMID: 33497524DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 12, 2018
First Posted
March 13, 2019
Study Start
September 26, 2019
Primary Completion
May 23, 2025
Study Completion
May 23, 2025
Last Updated
December 5, 2022
Record last verified: 2022-12
Data Sharing
- IPD Sharing
- Will not share