RECRUITING

A Long-term Follow-up Study of Gaucher Disease

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

A Long-term Follow-up Study of Gaucher Disease

Quick Facts

Study Start:2017-06-12
Study Completion:2027-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03190837

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:Not specified
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Diagnosis of Gaucher Disease
  1. * Not meeting Inclusion criteria

Contacts and Locations

Study Contact

Janet Blount
CONTACT
919-681-7962
janet.blount@duke.edu

Principal Investigator

Priya Kishnani, MD
PRINCIPAL_INVESTIGATOR
Duke University

Study Locations (Sites)

Duke University Medical Center
Durham, North Carolina, 27710
United States

Collaborators and Investigators

Sponsor: Duke University

  • Priya Kishnani, MD, PRINCIPAL_INVESTIGATOR, Duke University

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2017-06-12
Study Completion Date2027-01

Study Record Updates

Study Start Date2017-06-12
Study Completion Date2027-01

Terms related to this study

Keywords Provided by Researchers

  • gaucher
  • gaucher disease

Additional Relevant MeSH Terms

  • Gaucher Disease