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An Exploratory, Double-blind, Randomized, Placebo-controlled, Single-center, Two-way Cross-over Study With KH176 in Patients With the Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation and Clinical Signs of Mitochondrial Disease

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Trial Profile

An Exploratory, Double-blind, Randomized, Placebo-controlled, Single-center, Two-way Cross-over Study With KH176 in Patients With the Mitochondrial DNA tRNALeu(UUR) m.3243A>G Mutation and Clinical Signs of Mitochondrial Disease

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 10 Feb 2022

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At a glance

  • Drugs Sonlicromanol (Primary)
  • Indications Leber's hereditary optic atrophy; Leigh disease; MELAS syndrome; Mitochondrial disorders
  • Focus Proof of concept; Therapeutic Use
  • Acronyms KHENERGY
  • Sponsors Khondrion
  • Most Recent Events

    • 31 Jul 2018 According to a Khondrion media release, the company has received a grant of EUR 2.3M from the EU Horizon 2020 SME instrument program, which will be used for the execution of this clinical trial.
    • 30 Jul 2018 Results (n=20) published in the Clinical Pharmacology and Therapeutics.
    • 22 Feb 2018 Status changed from recruiting to completed.

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