Glycerol Tributyrate for MELAS Syndrome and Optic Neuropathy
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Do I need to stop my current medications to join the trial?
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.
Is there any evidence suggesting that this trial's treatment is likely to be safe?
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.
It is important to note that this treatment is in the early stages of human testing, meaning researchers are still learning about its safety. However, the study is carefully designed to minimize risks and ensure participant safety.12345Why are researchers excited about this trial's treatment for MELAS and optic neuropathy?
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.
What evidence suggests that glycerol tributyrate might be an effective treatment for MELAS and LHON-Plus?
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
Who Is on the Research Team?
Debra Regier, M.D., Ph.D.
Principal Investigator
Children's National Hospital; Children's National Rare Disease Institute
Wei-Liang Chen, M.D.
Principal Investigator
Children's National Research Institute
Anne Chiaramello, Ph.D.
Principal Investigator
George Washington University School of Medicine and Health Sciences
Are You a Good Fit for This Trial?
This trial is for individuals with MELAS or LHON-Plus, two types of mitochondrial diseases. Participants will be closely monitored and must have a stable clinical baseline established before starting the treatment. There's no placebo group due to ethical concerns, so all participants receive glycerol tributyrate.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Baseline Lead-in
Participants undergo a baseline lead-in phase to collect clinical, biochemical, molecular, and metabolic biomarkers.
Dose-Escalation
Participants receive escalating doses of glycerol tributyrate to determine the maximum tolerated dose (MTD).
Clinical
Participants receive a fixed participant-specific MTD of glycerol tributyrate to assess efficacy and safety.
Follow-up
Participants are monitored for adverse events and safety after the clinical phase.
What Are the Treatments Tested in This Trial?
Interventions
- Glycerol Tributyrate
Find a Clinic Near You
Who Is Running the Clinical Trial?
George Washington University
Lead Sponsor
Children's National Research Institute
Collaborator
National Center for Advancing Translational Sciences (NCATS)
Collaborator