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Gaucher Disease clinical trials in Pittsburgh, PA

5 studies for Gaucher Disease 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.

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A Gaucher Disease Gene Therapy Trial With FLT201

RecruitingPhase 3
  • UPMC Children's Hospital of Pittsburgh, Pittsburgh
Ages 18 and older · All sexes · Updated Sep 3, 2026 · NCT07223944

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

RecruitingObservational study
  • UPMC Children's Hospital of Pittsburgh- Site Number : 840023, Pittsburgh
All ages · All sexes · Updated Apr 14, 2026 · NCT00358943

Longitudinal Study of Neurodegenerative Disorders

RecruitingObservational study
  • UPMC Children's Hospital of Pittsburgh, Pittsburgh
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

RecruitingObservational study
  • UPMC Children's Hospital of Pittsburgh, Pittsburgh
2 Months – 60 Years · All sexes · Updated Jan 13, 2026 · NCT04528355

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

RecruitingPhase 2
  • UPMC Children's Hospital of Pittsburgh, Pittsburgh
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415

About Gaucher Disease

Gaucher disease is a rare, inherited disorder in which you do not have enough of an enzyme called glucocerebrosidase. Learn about the three types.

More about Gaucher Disease on MedlinePlus

Gaucher Disease 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.