Glycerol Tributyrate for MELAS Syndrome and Optic Neuropathy

This trial tests a new treatment called glycerol tributyrate for individuals with MELAS syndrome and LHON-Plus, rare mitochondrial diseases. The goal is to determine if this treatment is safe and improves symptoms such as muscle weakness or vision problems. Participants will take the treatment daily, with doses adjusted to find the optimal amount for each person. The trial is open to those with a confirmed diagnosis of MELAS or LHON-Plus who experience symptoms like muscle weakness or vision issues. As a Phase 1 and Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group.

The trial protocol does not specify whether you need to stop taking your current medications. It's best to discuss your specific situation with the study team.

Research is investigating the safety and effectiveness of glycerol tributyrate for treating MELAS syndrome and LHON-Plus, rare mitochondrial diseases. In completed studies, researchers have closely monitored any side effects. The trials aim to determine the safest dose for each participant, demonstrating a careful approach to ensure the treatment is well-tolerated.

Participants receive gradually increasing doses to assess how much they can safely handle before side effects appear. This process helps identify the optimal dose for each individual. Although the study is ongoing, no major safety concerns have been reported.

Researchers are excited about glycerol tributyrate for MELAS syndrome and optic neuropathy because it offers a novel approach compared to existing options like antioxidants and vitamin supplements. Unlike these standard treatments, glycerol tributyrate potentially targets the condition at a mitochondrial level, aiming to improve energy production and reduce oxidative stress directly. This treatment is administered orally, which could enhance patient compliance and comfort. By focusing on optimizing mitochondrial function, glycerol tributyrate might offer a more direct and effective way to manage these conditions, setting it apart from current therapies.

Research has shown that glycerol tributyrate might help with conditions related to MELAS syndrome and LHON-Plus, both mitochondrial disorders affecting the energy-producing parts of cells. In this trial, participants with MELAS syndrome will receive glycerol tributyrate in one treatment arm, while those with LHON-Plus will receive it in another. Glycerol tributyrate may improve the function of these energy-producing parts. Early findings suggest it could help with symptoms like stroke-like episodes in MELAS and vision problems in LHON-Plus. Although detailed information on its effectiveness in humans remains limited, the treatment is based on a strong understanding of how it might support energy production in cells. Ongoing studies are exploring this potential, and more data will help confirm its benefits.¹²⁴⁵⁶