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Gaucher Disease Outcome Survey (GOS)

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ClinicalTrials.gov Identifier: NCT03291223
Recruitment Status : Recruiting
First Posted : September 25, 2017
Last Update Posted : March 19, 2019
Sponsor:
Information provided by (Responsible Party):
Shire

Tracking Information
First Submitted Date August 8, 2017
First Posted Date September 25, 2017
Last Update Posted Date March 19, 2019
Actual Study Start Date July 27, 2010
Estimated Primary Completion Date December 1, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: September 19, 2017)
  • Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline to one year for up to 20 years ]
    Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
  • Number of Participants With Infusion-related Reactions (IRRs) [ Time Frame: Baseline to one year for up to 20 years ]
    An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.
  • Increase of Hemoglobin Concentration [ Time Frame: Baseline to one year for up to 20 years ]
    Hemoglobin concentration will be assessed.
  • Increase of Platelet Count [ Time Frame: Baseline to one year for up to 20 years ]
    Platelet count will be assessed.
  • Decrease in Liver Volume [ Time Frame: Baseline to one year for up to 20 years ]
    Liver volume will be assessed by abdominal imaging.
  • Decrease in Spleen Volume [ Time Frame: Baseline to one year for up to 20 years ]
    Spleen volume will be assessed by abdominal imaging.
  • Increase in Bone Mineral Density (BMD) [ Time Frame: Baseline to one year for up to 20 years ]
    Bone mineral density will be assessed.
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT03291223 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Gaucher Disease Outcome Survey (GOS)
Official Title Gaucher Disease Outcome Survey (GOS)
Brief Summary

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.

Detailed Description Not Provided
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 6 Months
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population GOS is a disease specific registry irrespective of treatment or treatment status, open to all patients of any age or sex with Gaucher disease of any type. Patients included may be those who are untreated, naive to therapy, individuals who are currently or have been previously treated with velaglucerase alfa (VPRIV), or individuals who have been receiving or are currently exposed to other treatments for Gaucher disease. There is no predefined sample size.
Condition Gaucher Disease
Intervention Not Provided
Study Groups/Cohorts GOS Participants
GOS is a disease specific registry open to all Gaucher patients irrespective of treatment status or type of treatment
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: September 19, 2017)
1257
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 1, 2020
Estimated Primary Completion Date December 1, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Patients of any age or gender with confirmed diagnosis (biochemical and/or genetic) of Gaucher disease
  • 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.

Exclusion Criteria:

- Patients currently enrolled in ongoing blinded clinical trials (drugs or devices; includes all blinded trials)

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03291223
Other Study ID Numbers GOS
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement Not Provided
Responsible Party Shire
Study Sponsor Shire
Collaborators Not Provided
Investigators
Study Director: Shire Study Physician Shire
PRS Account Shire
Verification Date March 2019