Registry for Gaucher Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to learn more about Gaucher disease, a rare genetic disorder affecting how the body processes certain fats. It observes patients with the condition to understand disease progression and the long-term effectiveness of current treatments, without introducing any new experimental drugs. The trial also focuses on pregnant women with Gaucher disease to gather information about pregnancy outcomes and infant growth. Individuals diagnosed with Gaucher disease, and who can provide medical documentation if pregnant, are suitable candidates for this trial. As an unphased study, this trial offers participants the opportunity to contribute to a deeper understanding of Gaucher disease and its impact on pregnancy and long-term health.
Will I have to stop taking my current medications?
The trial does not specify whether you need to stop taking your current medications. Since it's an observational study, you will likely continue your usual treatment as determined by your doctor.
Why are researchers excited about this trial?
Researchers are excited about the International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry because it offers a unique opportunity to gather comprehensive, real-world data on Gaucher disease, especially in pregnant patients. Unlike traditional treatments that focus on enzyme replacement or substrate reduction therapies, this observational program aims to understand the natural history and treatment outcomes of the disease in diverse populations. This registry could reveal important insights into how Gaucher disease affects different demographics, potentially leading to more personalized and effective treatment strategies in the future.
Who Is on the Research Team?
Study Director
Principal Investigator
Genzyme, a Sanofi Company
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observational Assessment
Participants undergo clinical assessments and receive care as determined by the patient's treating physician
Pregnancy Sub-registry
Tracks pregnancy outcomes, including complications and infant growth, in women with Gaucher disease during pregnancy
Follow-up
Participants are monitored for long-term effectiveness of imiglucerase and eliglustat
What Are the Treatments Tested in This Trial?
Interventions
- Observational Program
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris