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Prospective Natural History Study of POLG Disease

Recruitingn/aAges any - anyAllThe POLG Foundation
NCT07775872 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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

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📋 Eligibility rules (as published)
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.
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What it tests

  • Not listed
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Where

21 sites
University of California San Diego
San Diego, United States
Children's Hospital Colorado
Aurora, United States
Columbia University Irving Medical Center
New York, United States
Akron Children's Hospital
Akron, United States
Children's Hospital of Philadelphia-Philadelphia
Philadelphia, United States
Baylor Hospital
Dallas, United States
UTHealth Houston
Houston, United States
Prince of Wales Hospital, Sydney
Sydney, Australia

…and 13 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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