A Long-term Follow-up Study of Gaucher Disease

Part of paid clinical trials in Durham, North Carolina.

Sponsor
Duke University
Study ID
NCT03190837
Status
Recruiting
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Conditions

  • Gaucher Disease

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Jun 12, 2017
Status verified
Sep 2025
Primary completion
Jan 31, 2027
Completion
Jan 31, 2027

Study Design

Enrollment
200 participants (estimated)

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

Locations (1)

FacilityCityStateZIPSite coordinators
Duke University Medical CenterDurhamNorth Carolina27710-

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Duke University Medical Center· Durham, NC

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