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RECRUITINGNCT03190837

A Long-term Follow-up Study of Gaucher Disease

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),...

Lead sponsor: Duke University•1 U.S. locations•View source on ClinicalTrials.gov

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Contacts

Janet Blount

CONTACT

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

Sponsors

Lead Sponsor

Duke University

The study aims (aims a-c) will be accomplished by the systematic monitoring of health records. Health information will be extracted from medical records for patients followed at Duke. Medical records will be requested from treating physicians outside of Duke. The PI, (Dr. Kishnani) a recognized expert in Gaucher disease, is a consultant to the treating physician of virtually all subjects in this study. Subject health status will be monitored at least on an annual basis and as regularly as the medical information is obtained from the treating physician. Enrollment in this study will not alter the standard of care treatment for patients with Gaucher disease.

Age

All ages

Sex

ALL

Healthy Volunteers

No

Inclusion Criteria

•Diagnosis of Gaucher Disease

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: September 22, 2025Terms

Trial snapshot

StatusRECRUITING

Phase—

Enrollment200

Start dateJun 2017

Last updatedSep 22, 2025

Condition

Gaucher Disease

Locations shown

Duke University Medical Center

Durham, North Carolina

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: September 22, 2025Data synced to Clareo: May 10, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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