Prospective Natural History Study of POLG Disease

Brief summary (registry, in English)

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

What you commit to

• Study type: Observational
• Planned participants: 300
• From 17 Jun 2026 to the primary completion date, 17 Jun 2029
• 21 sites in 9 countries
• Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Who the study is looking for

Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (21)

Sites in registry order. Save your city in your profile to sort them by distance.