Actively Recruiting

Age: 4Years +
All Genders
Healthy Volunteers
NCT01532791

Natural History Study - Mitochondrial Disease

Led by Columbia University · Updated on 2026-01-23

300

Participants Needed

1

Research Sites

1147 weeks

Total Duration

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Sponsors

C

Columbia University

Lead Sponsor

E

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

Natural History Study - Mitochondrial Disease

Who Can Participate

Age: 4Years +
All Genders
Healthy Volunteers

Eligibility Criteria

Eligible

You may qualify if you...

  • Known carrier of the m.3243 A>G mitochondrial mutation, or maternally related to someone who carries the m.3243 A>G mitochondrial mutation
  • A family member who is not maternally related to someone who carries the m.3243 A>G mitochondrial mutation
Not Eligible

You will not qualify if you...

  • Younger than 4 years of age
  • No confirmed m.3243 A>G mitochondrial DNA mutation in the family.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Columbia University

New York, New York, United States, 10032

Actively Recruiting

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Research Team

K

Kris Engelstad, MS

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

2

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