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Rare Diseases clinical trials in St. Louis, MO

3 studies for Rare Diseases are looking for participants at sites in and around St. Louis (within about 30 miles). Open a study to see who can join and how to contact the study team.

Recruiting for a study here? Feature it at the top of this page — $99 for 30 days →

Clinical and Genetic Evaluation of Individuals With Undiagnosed Disorders Through the Undiagnosed Diseases Network

RecruitingObservational studyHealthy volunteers welcome
  • Washington University in St. Louis, St Louis
1 Month – 100 Years · All sexes · Updated Oct 2, 2026 · NCT02450851

Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

RecruitingPhase 2
  • Washington University in St. Louis, St Louis
Ages 18 and older · All sexes · Updated Jun 23, 2026 · NCT07123155

The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

RecruitingPhase 2
  • Washington University in St. Louis School of Medicine, St Louis
Ages 12 and older · All sexes · Updated Jul 1, 2025 · NCT04372615

About Rare Diseases

A rare disease is one that affects fewer than 200,000 people in the United States. There are nearly 7,000, affecting more than 25 million Americans.

More about Rare Diseases on MedlinePlus

Rare Diseases trials in other cities

Other studies in St. Louis

From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.