Clinical trial NCT03291223: Gaucher Disease Outcome Survey (GOS)
Official title: Gaucher Disease Outcome Survey (GOS)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Gaucher Disease
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2017-09-25
Enrollment: 1257
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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: 20 MAY 2020: The temporary enrollment stop of new patients into this study due to the COVID-10 pandemic has been lifted in one or more countries/sites, and the study is now again enrolling new patients. However, some countries/sites may still have paused the enrollment of new patients due to the pandemic.

24 APRIL 2020: Enrollment of new patients into this study has been paused due to the COVID-19 situation. The duration of this pause is dependent on the leveling and control of the COVID-19 pandemic.

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)

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline to one year for up to 20 years
measure: Number of Participants With Infusion-related Reactions (IRRs)
timeFrame: Baseline to one year for up to 20 years
measure: Increase of Hemoglobin Concentration
timeFrame: Baseline to one year for up to 20 years
measure: Increase of Platelet Count
timeFrame: Baseline to one year for up to 20 years
measure: Decrease in Liver Volume
timeFrame: Baseline to one year for up to 20 years
measure: Decrease in Spleen Volume
timeFrame: Baseline to one year for up to 20 years
measure: Increase in Bone Mineral Density (BMD)
timeFrame: Baseline to one year for up to 20 years
Secondary outcomes:
Other outcomes:
Locations:
city: Lexington
country: United States
facility: Central Contact
state: Massachusetts
Study officials:
affiliation: Shire
name: Shire Study Physician
role: STUDY_DIRECTOR
References: