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Recruiting NCT01532791

NCT01532791 Natural History Study - Mitochondrial Disease

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Clinical Trial Summary
NCT ID NCT01532791
Status Recruiting
Phase
Sponsor Columbia University
Condition MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Study Type OBSERVATIONAL
Enrollment 300 participants
Start Date 2004-07
Primary Completion 2026-07

Eligibility & Interventions

Sex All sexes
Min Age 4 Years
Max Age N/A
Study Type OBSERVATIONAL

Eligibility Fast-Check

Enter your details for a quick preliminary check. This does not replace medical advice.

What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 300 participants in total. It began in 2004-07 with a primary completion date of 2026-07.

⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.

Brief Summary

Carriers of the m.3242A\>G mutation often have clinical symptoms which can include migraines, seizures, strokes, hearing loss, balance issues, gastrointestinal issues, and many other symptoms. The investigators would like to learn more about these disorders and have designed a "Natural History Study" to monitor these conditions over time so that physicians and scientists can not only understand the problems that patients have, but work on developing treatments. The focus of the current work is to evaluate known mutation carriers of the m.3243A\>G (mitochondrial DNA) and their maternal relatives (carrier status not a requirement for participation). Paternal relatives will serve as controls. This study involves no treatment.

Eligibility Criteria

Inclusion Criteria: Known carrier of a the m.3243 A\>G mitochondrial mutation, ,or Maternally related to someone who carries the m.3243A\>G mitochondrial mutation. A family member who is not maternally related to someone who carries the m.3243A\>G mitochondrial mutation Exclusion Criteria: * Younger than 4 years of age * No confirmed m.3243 A\>G mitochondrial DNA mutation in the family.

Contact & Investigator

Central Contact

Kris Engelstad, MS

✉ ke4@cumc.columbia.edu

📞 2123056834

Principal Investigator

Michio Hirano, MD

PRINCIPAL INVESTIGATOR

mh29@cumc.columbia.edu

Frequently Asked Questions

Who can join the NCT01532791 clinical trial?

This trial is open to participants of all sexes, aged 4 Years or older, studying MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.

Is NCT01532791 currently recruiting?

Yes, NCT01532791 is actively recruiting participants. Contact the research team at ke4@cumc.columbia.edu for enrollment information.

Where is the NCT01532791 trial being conducted?

This trial is being conducted at New York, United States.

Who is sponsoring the NCT01532791 clinical trial?

NCT01532791 is sponsored by Columbia University. The principal investigator is Michio Hirano, MD at mh29@cumc.columbia.edu. The trial plans to enroll 300 participants.

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ClinicalMetric — Independent clinical trial intelligence platform. Not affiliated with NIH, ClinicalTrials.gov, the U.S. FDA, or any pharmaceutical company, hospital, or clinical research organization. Trial data is sourced from ClinicalTrials.gov for informational purposes only and does not constitute medical advice. Do not make any treatment, enrollment, or health decisions based solely on information found here — always consult a qualified healthcare professional. Full Disclaimer  ·  Last Reviewed: July 2026  ·  Data Methodology