Advancing Integrated Therapies for Gaucher Disease

Part of paid clinical trials in New Haven, Connecticut.

Sponsor
Yale University
Study ID
NCT07758816
Status
Recruiting
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Conditions

  • Gaucher Disease

Eligibility Criteria

Sex
ALL
Age
3 Months - 75 Years
Healthy Volunteers
Not accepted

Study Details

The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving standard medical care

Key Dates

First listed
Aug 11, 2026
Start date
May 6, 2026
Status verified
Aug 2026
Primary completion
Sep 30, 2030
Completion
Sep 30, 2030

Study Design

Enrollment
30 participants (estimated)

Arms

  • Arm: Neuronopathic Gaucher Disease
    Participants with GD2 or GD3 and documented pulmonary disease and/or massive lymphadenopathy.
  • Arm: Refractory Skeletal Disease
    Participants with GD1 or GD3, ages 10-75, with persistent skeletal disease despite long-term therapy.
  • Arm: Gaucher-Parkinson Overlap
    Adult participants only with Gaucher disease and clinical features of Parkinson disease.

Primary Outcome Measure

Change in pulmonary and lymphatic Gaucher disease burden [ Time Frame: Baseline, 6, 12, 24, and 36 months ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Yale New Haven Health SystemNew HavenConnecticut06520-

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By research site
Yale New Haven Health System· New Haven, CT

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