What is this study trying to find out about Gaucher disease?

This observational study aims to better understand how Gaucher disease progresses and varies among patients. It collects information to help guide treatment and improve patient care. The study is sponsored by Genzyme, a Sanofi Company, and involves tracking patient outcomes without testing new treatments.

Who may be eligible for this Gaucher disease clinical trial?

Anyone with a confirmed diagnosis of Gaucher disease can participate, regardless of age or gender. Pregnant women with Gaucher disease may also join the Pregnancy Sub-registry. Participants must provide proper consent and medical documentation. Please review eligibility criteria for more details.

Is there any cost to participate in this Gaucher disease clinical trial?

Study-related care, including all assessments and data collection, is provided at no cost. However, personal travel expenses or other costs related to visits may vary and are not guaranteed to be covered.

Actively Recruiting

All Genders

ID00358943

Gaucher Disease Registry Protocol

Led by Genzyme, a Sanofi Company · Updated on 2026-04-14

12000

Participants Needed

318

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are tracking the natural history and clinical outcomes of patients with Gaucher disease through the ICGG Gaucher Registry, an international, multi-center observational program. This registry does not involve any experimental treatments, but collects information to better understand the variability, progression, and identification of Gaucher disease, aiming to improve patient care and therapeutic guidance. It also evaluates the long-term use of treatments like imiglucerase and eliglustat. The study includes two groups patients with Gaucher disease who receive routine clinical assessments and standard care as determined by their physicians, and a Pregnancy Sub-registry for women with Gaucher disease who are pregnant or have been pregnant. The Pregnancy Sub-registry collects information on pregnancy outcomes, complications, and infant growth up to 36 months postpartum, regardless of whether the women receive disease-specific therapy. Participants undergo clinical assessments and receive care according to their treating physicians decisions. Data collected includes medical history, pregnancy and birth details for the sub-registry, and patient outcomes over time. The primary goals are to provide recommendations for monitoring Gaucher disease patients, report outcomes to optimize care, and track pregnancy and infant growth outcomes. This ongoing registry allows long-term follow-up without experimental interventions.

CONDITIONS

Brief Title

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Who Can Participate

All Genders

Eligibility Criteria

You may qualify if you...

All patients with a confirmed diagnosis of Gaucher disease, defined by documented b2-glucocerebrosidase deficiency or gene mutation, are eligible.
Patients must provide appropriate authorization to participate.
For the Pregnancy Sub-registry, patients must be enrolled in the ICGG Gaucher Registry.
Pregnant women or those who have been pregnant with medical documentation are eligible for the Sub-registry.
Patients must provide signed informed consent and authorization forms before data collection for the Sub-registry.

You will not qualify if you...

There are no exclusion criteria for participation in the ICGG Gaucher Registry and Pregnancy Sub-registry.

Research Team

Trial Transparency email recommended (Toll free number for US & Canada)

ICGG Gaucher Registry HelpLine

Not the Right Trial for You?

Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.

Already have an account? Log in here