NCT07758816Recruiting
Advancing Integrated Therapies for Gaucher Disease
Advancing Integrated Therapies for Gaucher Disease: Neuronopathic Innovation and Combination Strategies for Refractory Skeletal Disease
Source: ClinicalTrials.gov · Last updated 11 Aug 2026
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Study type
- Observational
- Enrollment
- 30 (estimated)
- First submitted
- 5 Aug 2026
- Start date
- 6 May 2026
- Primary completion date
- Sept 2030
- Lead sponsor
- Yale University
- Diseases
- Gaucher disease
- NCT number
- NCT07758816
What you commit to
- Study type: Observational
- Planned participants: 30
- From 6 May 2026 to the primary completion date, Sept 2030
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Any condition that, in the investigator's judgment, precludes safe participation
Other
- Aim 1
- Inability to comply with observational follow-up
Who the study is looking for
- Aim 1
- 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
- Ability to provide informed consent (or parental consent with assent as appropriate)
- Aim2
- ages 10-75
- persistent skeletal disease despite long-term therapy
- Aim3
- Gaucher disease and clinical features of Parkinson disease
- Ability to provide informed consent
Age in the registry: 3 months to 75 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Yale-New Haven Health System Coordinator of this site? Confirm its status | New Haven | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.