RECRUITING

Gaucher Disease Outcome Survey (GOS)

Conditions

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 Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.

Official Title

Gaucher Disease Outcome Survey (GOS)

Quick Facts

Study Start:2010-07-27
Study Completion:2025-01-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03291223

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:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease
  2. * Signed and dated written informed consent from the patient or, for patients aged \<18 years (\<16 years in the United Kingdom \[UK\]), their parent and/or legally authorized representatives (LAR), and assent of the minor where applicable. Legally authorized representatives are also applicable for cognitively impaired patients.
  1. Pregnancy or breastfeeding
  2. Severe psychiatric disorders
  3. Active substance abuse
  4. Unstable medical conditions
  5. Inability to comply with study requirements

Contacts and Locations

Study Contact

Shire Contact
CONTACT
+1 866 842 5335
ClinicalTransparency@shire.com

Principal Investigator

Shire Study Physician
STUDY_DIRECTOR
Shire

Study Locations (Sites)

Central Contact
Lexington, Massachusetts, 02421
United States

Collaborators and Investigators

Sponsor: Shire

  • Shire Study Physician, STUDY_DIRECTOR, Shire

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 Date2010-07-27
Study Completion Date2025-01-03

Study Record Updates

Study Start Date2010-07-27
Study Completion Date2025-01-03

Terms related to this study

Additional Relevant MeSH Terms

  • Gaucher Disease