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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")

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Trial Profile

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")

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 17 Jul 2023

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At a glance

  • Drugs EDIT 101 (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions; First in man; Proof of concept
  • Acronyms Brilliance
  • Sponsors AbbVie; Allergan; Editas Medicine
  • Most Recent Events

    • 16 Jul 2023 This trial has been discontinued in Netherlands, according to a European Clinical Trials Database record.
    • 02 Dec 2022 Planned End Date changed from 22 Mar 2024 to 23 May 2025.
    • 02 Dec 2022 Planned primary completion date changed from 22 Mar 2024 to 23 May 2025.

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