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Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of AGN-151587 (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")

Trial Profile

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Efficacy of AGN-151587 (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: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 23 Nov 2019

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At a glance

  • Drugs EDIT 101 (Primary)
  • Indications Leber congenital amaurosis
  • Focus Adverse reactions
  • Acronyms Brilliance
  • Sponsors Allergan
  • Most Recent Events

    • 12 Nov 2019 According to an Allergan media release, dosing of first patient in this trial is expected in early 2020.
    • 25 Jul 2019 According to an Allergan media release, sites in the U.S. are currently enrolling patients for the trial, including Massachusetts Eye and Ear, an international center for treatment and research and a teaching hospital of Harvard Medical School.
    • 22 Jul 2019 Status changed from not yet recruiting to recruiting.
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