๐Ÿ›๏ธ Verified NIH Record โ€ข Updated 2026-08-18

Prospective Natural History Study of POLG Disease (NCT07775872)

Global Prospective Natural History Study of POLG Disease

๐ŸŸก Not Yet Recruiting Interventional Protocol ๐Ÿ“Š Observational Clinical Research Sponsor: The POLG Foundation

๐Ÿฉบ Plain-English Summary (8th-Grade Level)

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

Who can join: Review the eligibility criteria below.

Location: University of California San Diego โ€” San Diego, California

Age range: Not specified to 75 Years

๐Ÿ“‹ "Do I Qualify?" โ€” 1-Minute Self Screener

Instant Checklist

Full eligibility criteria are available on the official NIH protocol. View the criteria โ†—

๐Ÿ“ All Participating Trial Locations (6 Sites)

  • University of California San Diego โ€” San Diego, California
  • Children's Hospital Colorado โ€” Aurora, Colorado
  • Columbia University Irving Medical Center โ€” New York, New York
  • Akron Children's Hospital โ€” Akron, Ohio
  • Baylor Hospital โ€” Dallas, Texas
  • UTHealth Houston โ€” Houston, Texas

๐Ÿ“ž Contact the Study Coordinator

Phone: (571) 609-4617

Email: pioneer@polgfoundation.org

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

๐Ÿ’ฐ Cost, Insurance & Patient Rights

  • 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
  • Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
  • Voluntary Participation: You may withdraw at any time without affecting your standard medical care.

๐Ÿ’ก How to Participate

  1. Check the checklist above to see if you may qualify.
  2. Contact the study coordinator using the phone/email above.
  3. Ask about the visit schedule, what is covered at no cost, and any travel support.
  4. Confirm with your own doctor before making a decision.
๐Ÿ›๏ธ View Official NIH Record โ†—