Recruiting

Observational StudyA Long-term Follow-up Study of Gaucher Disease

Sponsor:

Duke University

Code:

NCT03190837

Conditions

Gaucher Disease

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

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.

Conditions

Gaucher Disease

Study ID

NCT03190837

Start date

Jun 12, 2017

Status verified date

Sep, 2026

Completion date

Jan, 2028

Anticipated

Primary completion date

Jan, 2028

Anticipated

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

Diagnosis of Gaucher Disease

Exclusion Criteria:

Not meeting Inclusion criteria

Study Design

Enrollment

200 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

Document effects of Gaucher disease in different systems of body, including nervous system, liver, and spleen. [ Time Frame: 10 years ]

Central Contacts and Locations

Central contacts

Janet Blount

919-681-7962 janet.blount@duke.edu

Locations

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

More Information

Sponsor

Duke University

Last update posted

Sep 3, 2026

Last verified

Sep, 2026

Keywords

gaucher
gaucher disease

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Duke University on 2026-09-03.