Home › Gaucher Disease › NCT03190837

A Long-term Follow-up Study of Gaucher Disease

Who can join

All ages · All sexes

Inclusion Criteria:

* Diagnosis of Gaucher Disease

Exclusion Criteria:

* Not meeting Inclusion criteria

About the study

The study aims are to: a) identify the long-term natural history of Gaucher disease, b) evaluate long-term treatment efficacy of enzyme replacement therapy (ERT) and substrate reduction therapy (SRT), and c) identify potential long-term complications of this disorder. These aims will be accomplished through long-term record review of individuals with all three types of Gaucher disease.

Sponsor: Duke University · Participants: 200 · Started: Jun 12, 2017

Contact the study team

• Janet Blount · Phone: 919-681-7962

Official record on ClinicalTrials.gov — NCT03190837

Locations in the U.S.

Conditions

• Gaucher Disease

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.