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Gaucher Disease clinical trials recruiting in the U.S.
14 studies for Gaucher Disease are recruiting participants at sites across the United States.
Gaucher disease is a rare, inherited disorder in which you do not have enough of an enzyme called glucocerebrosidase. Learn about the three types.
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Recently updated studies
Gaucher Disease Outcome Survey (GOS)
RecruitingObservational study
All ages · All sexes · Updated Sep 15, 2026 · NCT03291223A Gaucher Disease Gene Therapy Trial With FLT201
RecruitingPhase 3
Ages 18 and older · All sexes · Updated Sep 3, 2026 · NCT07223944A Long-term Follow-up Study of Gaucher Disease
RecruitingObservational study
All ages · All sexes · Updated Sep 3, 2026 · NCT03190837Advancing Integrated Therapies for Gaucher Disease
RecruitingObservational study
3 Months – 75 Years · All sexes · Updated Aug 11, 2026 · NCT07758816MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
RecruitingObservational study
Ages 18 to 80 · All sexes · Updated Aug 4, 2026 · NCT05586243Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
RecruitingObservational study
Ages 18 to 75 · All sexes · Updated Jul 13, 2026 · NCT02437396International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
RecruitingObservational study
All ages · All sexes · Updated Apr 14, 2026 · NCT00358943Registry of Patients Diagnosed With Lysosomal Storage Diseases
RecruitingObservational study
Up to age 64 · All sexes · Updated Apr 8, 2026 · NCT05619900Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
RecruitingObservational study
Ages 18 to 80 · All sexes · Updated Apr 6, 2026 · NCT05843552Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma
RecruitingPhase 1
18 Months · All sexes · Updated Mar 27, 2026 · NCT03721068PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
RecruitingPhase 1
Ages 18 to 50 · Women · Updated Mar 17, 2026 · NCT04532047Longitudinal Study of Neurodegenerative Disorders
RecruitingObservational study
All ages · All sexes · Updated Feb 9, 2026 · NCT03333200Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
RecruitingObservational study
2 Months – 60 Years · All sexes · Updated Jan 13, 2026 · NCT04528355Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
RecruitingPhase 2
2 Months – 55 Years · All sexes · Updated Dec 15, 2025 · NCT01962415From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.