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NCT06792500 · ClinicalTrials.gov registry record · Phase 1
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)
A Phase 1 study of MELAS Syndrome and Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus), sponsored by George Washington University.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
- 1
- Study location
NCT06792500 is a Phase 1 study of MELAS Syndrome and Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus) that is actively recruiting participants, run by George Washington University. The registered enrollment target is 24 participants. The trial reports 1 study location across 1 state.
The verdict
NCT06792500, a Phase 1 study of MELAS Syndrome and Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus), is actively recruiting participants, sponsored by George Washington University.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
- 1
- Study location
Study Summary
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, eliminating the need for a placebo. Considering the high clinical heterogeneity among participants affected by MELAS or LHON-Plus and some clinical divergence between MELAS and LHON-Plus, this strategy is beneficial to every enrolled participants, as each will receive the investigational drug, glycerol tributyrate. In addition, this approach will determine the subject-specific maximal optimized dose in a personalized medicine-based approach. After approval of the IRB protocol from the Institutional Review Board Data and signed consent form from all participants, this investigational basket clinical trial has three phases spanning over 20 months: * A baseline lead-in phase (2 months) to collect participant-specific baseline for clinical, biochemical, molecular and metabolic biomarkers that will be monitored throughout the subsequent dose-escalation and clinical phases. * A dose-escalation ph
Conditions Studied
Interventions
- DRUG Glycerol Tributyrate
Study Locations (1)
District of Columbia
- Children's National Hospital - Washington D.C.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2026-04 |
| Est. Completion | 2027-10 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06792500
The ClinicalTrials.gov registry entry for NCT06792500 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 24 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is George Washington University, which has 133 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with MELAS Syndrome appearing as the primary indexed condition, and to 1 intervention - of which Glycerol Tributyrate is the first listed.
NCT06792500 reports 1 study location spanning 1 distinct geographic area - top geographies include District of Columbia.
Frequently Asked Questions
What is clinical trial NCT06792500 about?
NCT06792500 is a clinical study titled "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)". 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 ...
What is the current status of trial NCT06792500?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2026-04. Estimated completion is 2027-10.
What conditions does trial NCT06792500 study?
This clinical trial studies the following conditions: MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus).
What interventions are being tested in trial NCT06792500?
The interventions under investigation include: Glycerol Tributyrate (DRUG).
Who is sponsoring clinical trial NCT06792500?
This trial is sponsored by George Washington University, which has 133 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06792500 being conducted?
This trial has 1 study location across District of Columbia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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