NCT07775872 Prospective Natural History Study of POLG Disease
| NCT ID | NCT07775872 |
| Status | Recruiting |
| Phase | — |
| Sponsor | The POLG Foundation |
| Condition | PolG, Primary Mitochondrial Myopathy |
| Study Type | OBSERVATIONAL |
| Enrollment | 300 participants |
| Start Date | 2026-06-17 |
| Primary Completion | 2029-06-17 |
Eligibility & Interventions
Eligibility Fast-Check
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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 2026-06-17 with a primary completion date of 2029-06-17.
⚠ 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
The PIONEER study is a prospective, natural history study dedicated to characterizing the clinical progression of POLG-related disorders. The research aims to bridge the gap between genetic diagnosis and drug development by mapping how these rare mitochondrial conditions evolve over time. By observing the disease's natural trajectory through a multi-center approach, The study identifies critical clinical milestones that serve as a foundation for evaluating therapeutic efficacy and future therapeutic interventions
Eligibility Criteria
Inclusion Criteria: * male \& female from age 0 to 75. * A genetically confirmed POLG -associated disorder based on both phenotype and genotype is required. * Parental/guardian permission (informed consent) and if appropriate with child assent. Exclusion Criteria: * Diagnosis of mitochondrial disorder other than POLG * Subject with POLG Variant of unknown significance or benign variant. * Parents/guardians or subjects who, in the opinion of the investigator, may be non-compliant with the study schedules or procedures. * Subjects unable or unwilling to provide informed consent. * History of or current clinically important condition other than what is related to the PMD which, in the opinion of the Investigator will confound the results of the NHS.
Contact & Investigator
Amel Kaara
PRINCIPAL INVESTIGATOR
Harvard Medical School & Massachusetts General Hospital
Frequently Asked Questions
Who can join the NCT07775872 clinical trial?
This trial is open to participants of all sexes, studying PolG, Primary Mitochondrial Myopathy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07775872 currently recruiting?
Yes, NCT07775872 is actively recruiting participants. Contact the research team at pioneer@polgfoundation.org for enrollment information.
Where is the NCT07775872 trial being conducted?
This trial is being conducted at San Diego, United States, Aurora, United States, New York, United States, Akron, United States and 11 additional locations.
Who is sponsoring the NCT07775872 clinical trial?
NCT07775872 is sponsored by The POLG Foundation. The principal investigator is Amel Kaara at Harvard Medical School & Massachusetts General Hospital. The trial plans to enroll 300 participants.