Clinical trial
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")
NCT03872479 is registered in ClinicalTrials.gov with Editas Medicine, Inc. as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- 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")
- Lead sponsor
- Editas Medicine, Inc.
- Phase
- Phase 1/2
- Overall status
- Unknown status
- Start date
- September 26, 2019
- Completion date
- May 23, 2025
- Enrollment
- 34 (estimated)
- Sites
- 5
Conditions
As the registry lists them; a condition with its own page is linked.
eye diseases, hereditary, eye disorders congenital, inherited retinal dystrophies, leber congenital amaurosis 10, retinal degeneration, retinal disease, vision disorders