Melas Syndrome
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CLINICAL_TRIALS research · 2026-02-09Clinical Trial Update: A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus) [Phase 1, Phase 2]
This is a basket clinical study evaluating glycerol tributyrate in patients with MELAS syndrome or Leber's hereditary optic neuropathy, curr…
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CLINICAL_TRIALS research · 2026-01-23Clinical Trial Update: Natural History Study - Mitochondrial Disease
Columbia University is sponsoring a natural history study for individuals with MELAS and related m.3243 A>G mitochondrial DNA mutations, cur…
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CLINICAL_TRIALS research · 2025-09-09Clinical Trial Update: A Study to Assess TTI-0102 vs Placebo in MELAS Patients [Phase 2]
A Phase 2 study evaluating TTI-0102 versus placebo in MELAS patients is currently recruiting. The sponsor is Thiogenesis Therapeutics, Inc.
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CLINICAL_TRIALS research · 2025-06-05Clinical Trial Update: Global Registry and Natural History Study for Mitochondrial Disorders
LMU Klinikum is conducting a global registry and natural history study to collect data on mitochondrial disorders, and the study is currentl…
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CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: The KHENERGYC Study [Phase 2]
Phase 2 KHENERGYC study for mitochondrial diseases is currently suspended.
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CLINICAL_TRIALS trial_result · 2024-06-26Clinical Trial Update: L-Citrulline Dose Finding Safety Study in MELAS [Phase 1]
Phase 1 safety and dose-finding study of L-Citrulline in MELAS syndrome has completed, assessing tolerability and safe dose ranges.
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CLINICAL_TRIALS trial_result · 2022-08-31Clinical Trial Update: The KHENERGYZE Study [Phase 2]
Phase 2 trial update for the KHENERGYZE study investigating a treatment for mitochondrial diseases and mitochondrial myopathies (including M…
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CLINICAL_TRIALS trial_result · 2018-04-27Clinical Trial Update: Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers [Phase 1]
A Phase 1 study evaluated safety, tolerability, and pharmacokinetics of KL1333 in healthy male volunteers; the trial has completed.
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CLINICAL_TRIALS trial_result · 2017-04-21Clinical Trial Update: Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS) [Phase 4]
Phase 4 trial evaluating the immune response to influenza vaccination in patients with MELAS syndrome; results were posted in 2017.
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CLINICAL_TRIALS trial_result · 2016-08-10Clinical Trial Update: Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes [Phase 2]
A Phase 2 trial evaluating idebenone for MELAS syndrome has completed and results were posted in 2016.
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CLINICAL_TRIALS trial_result · 2016-04-05Clinical Trial Update: Nitric Oxide Production in MELAS Syndrome [EARLY_Phase 1]
An early-phase 1 trial evaluating nitric oxide production in MELAS syndrome, sponsored by Baylor College of Medicine, has completed.
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CLINICAL_TRIALS trial_result · 2014-04-15Clinical Trial Update: Mitochondrial nt3243 A>G Mutation in Taiwan
This entry reports a clinical trial update in Taiwan focusing on the mitochondrial nt3243 A>G mutation in patients with MELAS syndrome and m…
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CLINICAL_TRIALS trial_result · 2013-12-17Clinical Trial Update: L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome [Phase 2]
Phase 2 clinical trial evaluating L-arginine for endothelium-dependent vasodilation and mitochondrial metabolism in MELAS syndrome has compl…
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CLINICAL_TRIALS trial_result · 2012-02-06Clinical Trial Update: Ketones & Mitochondrial Heteroplasmy [EARLY_Phase 1]
An early-phase 1 trial evaluating ketones and mitochondrial heteroplasmy has completed, studying MELAS syndrome and other mitochondrial dise…
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CLINICAL_TRIALS trial_result · 2008-07-10Clinical Trial Update: Study of the Metabolism of Pyruvate and Related Problems in Patients With Lactic Acidemia
This update notes the completion of a clinical trial studying pyruvate metabolism in patients with lactic acidosis, including individuals wi…