RecruitingNCT07775872

Prospective Natural History Study of POLG Disease

Global Prospective Natural History Study of POLG Disease

Sponsor

The POLG Foundation

Enrollment

300 participants

Start Date

Jun 17, 2026

Study Type

OBSERVATIONAL

Conditions

PolG, Primary Mitochondrial Myopathy

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

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a new treatment for people with polg, primary mitochondrial myopathy. The study is currently recruiting participants at 21 locations.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

Locations(21)

University of California San Diego

San Diego, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Columbia University Irving Medical Center

New York, New York, United States

Akron Children's Hospital

Akron, Ohio, United States

Children's Hospital of Philadelphia-Philadelphia

Philadelphia, Pennsylvania, United States

Baylor Hospital

Dallas, Texas, United States

UTHealth Houston

Houston, Texas, United States

Prince of Wales Hospital, Sydney

Sydney, New South Wales, Australia

Copenhagen University Hospital- Copenhagen

Copenhagen, Capital Region of Denmark, Denmark

Children's Hospital, Helsinki University Hospital-Helsinki

Helsinki, Uusimaa, Finland

Oulu University Hospital-Oulu

Helsinki, Uusimaa, Finland

Maastricht University-Maastricht

Maastricht, Limburg, Netherlands

Oslo University Hospital

Oslo, Oslo, Norway

University Hospital of North Norway - Tromsø

Tromsø, Troms, Norway

St. Olav's Hospital-Trondheim

Trondheim, Trøndelag, Norway

Haukeland University Hospital-Bergen

Bergen, Vestland, Norway

Hospital 12 de Octubre- Madrid

Madrid, Madrid, Spain

Karolinska University Hospital

Solna, Stockholm County, Sweden

The Queen Silvia Children's Hospital, University of Gothenburg

Gothenburg, Västra Götaland County, Sweden

Addenbrooke's Hospital, Cambridge University Hospitals

Cambridge, Cambridgeshire, United Kingdom

Great Ormond Street Hospital

London, Central London, United Kingdom

View Full Details on ClinicalTrials.gov

For the most up-to-date information, visit the official listing.

Visit

NCT07775872