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NCT05241262 · ClinicalTrials.gov registry record · Phase 1
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
A Phase 1 study of Mitochondrial Disease, sponsored by Michio Hirano, MD.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 18
- Enrollment target
- 1
- Study location
NCT05241262: Recruiting Phase 1 study of Mitochondrial Disease, sponsored by Michio Hirano, MD.
NCT05241262 is a Phase 1 study of Mitochondrial Disease that is actively recruiting participants, run by Michio Hirano, MD. The registered enrollment target is 18 participants, below the 184-participant average among 5 other Mitochondrial Disease trials with a reported enrollment target (90% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05241262, a Phase 1 study of Mitochondrial Disease, is actively recruiting participants, sponsored by Michio Hirano, MD.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 18 participants
- Enrollment target
- 1
- Study location
Study Summary
N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life. A group of 18 participants will take either 1800, 3600 or 5400 mg per day of N-acetylcysteine (NAC) for 3 months in this dose escalation study. The investigators want to determine first if the 3600 mg dose per day is safe and might provide some efficacy. If the 3600 mg dose is safe, then additional participants will be treated with 5400 mg per day of NAC, for up to a total of 18 participants. If the 3600 mg per day dose is unsafe, then participants will be treated with the 1800 mg per day dose. Data from this pilot study will be used to determine the most safe and effective dose of NAC for a future clinical trial.
Primary Outcome
Establish maximum tolerated dose of NAC by initiating dosing at one level, then increasing or decreasing dose based on toxicity. The MTD is defined as the dose associated with a target probability of dose limiting toxicity (DLT) of 0.10.
Conditions Studied
Interventions
- DRUG N-Acetylcysteine
Study Locations (1)
New York
- Columbia University Irving Medical Center - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 18 participants |
| Start Date | 2023-07-06 |
| Est. Completion | 2026-03 |
| Phase | Phase 1 |
What NCT05241262 shows while recruiting
NCT05241262 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 18 participants, a relatively small participant target, below the 184-participant average among 5 other Mitochondrial Disease trials with a reported enrollment target (90% lower).
The record links to 1 condition, with Mitochondrial Disease appearing as the primary indexed condition, and to 1 intervention - of which N-Acetylcysteine is the first listed.
NCT05241262 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT05241262 about?
NCT05241262 is a clinical study titled "Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels". N-Acetylcysteine (NAC), an anti-oxidant, will be studied to investigate the effects on brain glutathione levels, cognitive skills, motor skills, and quality of life. A group of 18 participants will take either 1800, 3600 or 5400 mg per day of N-acetylcysteine (NAC) for 3 months in this dose escalat...
What is the current status of trial NCT05241262?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 18 participants. The study started on 2023-07-06. Estimated completion is 2026-03.
What conditions does trial NCT05241262 study?
This clinical trial studies the following conditions: Mitochondrial Disease.
What interventions are being tested in trial NCT05241262?
The interventions under investigation include: N-Acetylcysteine (DRUG).
Who is sponsoring clinical trial NCT05241262?
This trial is sponsored by Michio Hirano, MD, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05241262 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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