RECRUITING

Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Multiple Ascending Phase 1 Dose Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

Quick Facts

Study Start:2023-07-06
Study Completion:2025-04
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05241262

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 80 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Ages 18-80 years
  2. * Low brain glutathione (GSH) levels as determined by magnetic resonance spectroscopic imaging (MRSI)
  3. * Individuals who carry, or are suspected of carrying the m.3243A\>G mitochondrial mutation (genetic confirmation of mutation required prior to initiation of NAC)
  1. * Individuals with normal brain glutathione levels
  2. * Pregnant or lactating individuals
  3. * Medically unstable as determined by the Principal Investigator
  4. * Allergy to NAC or other sulfur-containing drug
  5. * Inability to adhere to study protocol

Contacts and Locations

Study Contact

Kris Engelstad, MS, CGC
CONTACT
212-342-5767
ke4@cumc.columbia.edu

Principal Investigator

Michio Hirano, MD
PRINCIPAL_INVESTIGATOR
Columbia University

Study Locations (Sites)

Columbia University Irving Medical Center
New York, New York, 10032
United States

Collaborators and Investigators

Sponsor: Michio Hirano, MD

  • Michio Hirano, MD, PRINCIPAL_INVESTIGATOR, Columbia University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-07-06
Study Completion Date2025-04

Study Record Updates

Study Start Date2023-07-06
Study Completion Date2025-04

Terms related to this study

Keywords Provided by Researchers

  • m.3243A>G
  • MELAS
  • diabetes
  • deafness

Additional Relevant MeSH Terms

  • Mitochondrial Disease