Gaucher Disease Outcome Survey (GOS)

Condition(s) studied

Study 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.

Eligibility

Primary outcome measure(s)

• Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) — 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) — 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 — Baseline to one year for up to 20 years
  Hemoglobin concentration will be assessed.
• Increase of Platelet Count — Baseline to one year for up to 20 years
  Platelet count will be assessed.
• Decrease in Liver Volume — Baseline to one year for up to 20 years
  Liver volume will be assessed by abdominal imaging.
• Decrease in Spleen Volume — Baseline to one year for up to 20 years
  Spleen volume will be assessed by abdominal imaging.
• Increase in Bone Mineral Density (BMD) — Baseline to one year for up to 20 years
  Bone mineral density will be assessed.

Trial sites (1)

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