Phase 1/2Not yet recruiting
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)
Investigational Study of Glycerol Tributyrate in MELAS and LHON-Plus
- Sponsor
- George Washington University (Academic or other)
- Enrolment
- 24 planned
- Conditions
- MELAS Syndrome; Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
- Interventions
- Glycerol Tributyrate
- Ages
- adults, older adults
- Registry
- NCT06792500
Full record on ClinicalTrials.gov
From the registry
This is a parallel arm non-randomized dose-escalation, open-label basket exploratory phase 1 clinical trial where Mitochondrial encephalopathy, lactic acidosis, stroke-like episodes (MELAS) and Leber's hereditary optic neuropathy-Plus (LHON-Plus) participants will undergo simultaneous enrollment in two disease-based arms and receive daily oral doses of glycerol tributyrate to assess its safety and potential for efficacy using clinical, biochemical, and molecular evidence. This study will utilize a two-month baseline lead-in phase to establish and document the clinical baseline for each participant in both arms in order to compare the molecular and clinical parameters. This is clinically relevant in light of the high clinical heterogeneity among subjects affected by the same mitochondrial disease (MELAS or LHON-Plus). For ethical concerns prompted by the lack of treatment for these two intractable and progressive mitochondrial diseases, there will not be a placebo control group. Thus, each participant will act as their own control and receive oral doses of glycerol tributyrate,…
Primary outcomes
- Assessment of Dose Safety for Glycerol Tributyrate
- Potential Efficacy of Glycerol Tributyrate on the oxidative phsophorylation metabolism
History
Study completion expected
Primary completion expected
Expected to start
Last registry update
Registered