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Massive global registry launched to unlock secrets of gaucher disease

NCT ID NCT03291223

Recruiting now Knowledge-focused Sponsor: Shire Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study is a long-term registry that follows over 1,200 people with Gaucher disease worldwide. It collects real-world data on how the disease progresses and how different treatments, including velaglucerase alfa, work over time. No new drugs are being tested; instead, participants receive their usual care while researchers monitor safety and effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

What this could lead to
If successful, this registry could provide better evidence for managing Gaucher disease and improve understanding of its long-term course.
What could go wrong
As an observational study, it does not test a new treatment, so it cannot directly lead to a cure or new therapy. Results may be limited by differences in patient care and reporting.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Gaucher disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GAUCHER DISEASE

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Central Contact

    RECRUITING

    Lexington, Massachusetts, 02421, United States

    Contact

    Contact Email: •••••@•••••

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