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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: 20 Mar 2020

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At a glance

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

    • 04 Mar 2020 According to an Allergan media release, first patient has been dosed in the trial, at Oregon Health & Science University (OHSU) Casey Eye Institute. Mark Pennesi, M.D., Ph.D., Associate Professor of Ophthalmology, Kenneth C. Swan Endowed Professor, Division Chief, Paul H. Casey Ophthalmic Genetics, Casey Eye Institute, Oregon Health & Science University, Principal Investigator and enrolling physician of the first patient treated.
    • 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.
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