Advancing Integrated Therapies for Gaucher Disease
ConditionGaucher Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age3-75
SponsorYale University
About this trial
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
Eligibility criteria
Qualifiers
Confirmed diagnosis of GD2 or GD3 based on genotype and phenotype
Evidence of pulmonary infiltrative disease and/or mediastinal or mesenteric lymphadenopathy
Receiving or eligible for standard-of-care therapy
Age ≥ 3 months
Disqualifiers
Inability to comply with observational follow-up
Any condition that, in the investigator's judgment, precludes safe participation
Trial design
Treatments tested in this trial
- Not listed
Trial groups
30 Participants
are grouped into 3 trial groupsSponsors and collaborators
Yale University
Lead sponsor
Sanofi-Yale External Research Collaboration
Collaborator
Source ClinicalTrials.gov