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Rare Diseases clinical trials in Pittsburgh, PA

3 studies for Rare Diseases are looking for participants at sites in and around Pittsburgh (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 →

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

RecruitingPhase 2
  • University of Pittsburgh School of Medicine, Pittsburgh
Ages 18 and older · All sexes · Updated Jun 23, 2026 · NCT07123155

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

RecruitingPhase 1/2
  • University of Pittsburg, Pittsburgh
Ages 25 to 65 · All sexes · Updated May 5, 2026 · NCT06826612

The ExTINGUISH Trial of Inebilizumab in NMDAR Encephalitis

RecruitingPhase 2
  • University of Pittsburgh, Pittsburgh
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 Pittsburgh

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