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

This study, called the ICGG Gaucher Registry, is an ongoing international project that keeps track of how Gaucher disease affects people in their daily lives. Doctors collect information on patients' health and care as part of their routine check-ups. Nobody receives experimental treatments; it's all about observing real-world outcomes. The main goals are to better understand Gaucher disease, improve patient care guidelines, learn more about different types of patients, and see how long-term medicines like imiglucerase and eliglustat work. There's also a special part of the study for pregnant women with Gaucher disease. This helps doctors understand how pregnancy affects women with Gaucher disease and how their babies grow and develop after birth.

At a glance

Status
Recruiting
Sponsor
Genzyme, a Sanofi Company
Enrolment target
12,000
Start
01 Apr 1991
Estimated completion
31 Jan 2034

What is this study about?

Imagine a big, ongoing health diary for people with Gaucher disease from around the world. That's essentially what the International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry is. It's not a study where you try new medicines; instead, doctors simply record information about your health and the care you're already receiving as part of your regular check-ups. The main idea is to gather a lot of information over time to help doctors and researchers understand Gaucher disease better. This includes seeing how the condition can differ from person to person, how it progresses, and how current treatments are working in the long run.

The information collected helps medical experts create better recommendations for monitoring patients and provide reports that can improve overall patient care. Think of it as a shared learning experience for the global medical community. It also helps to understand the full range of people living with Gaucher disease and to evaluate how well certain long-term medications, like imiglucerase and eliglustat, are truly working over many years.

There's also a special part of this registry called the Gaucher Pregnancy Sub-registry. This is specifically for women with Gaucher disease who are pregnant or have been pregnant. The goal here is to carefully track how pregnancies progress, any complications that might arise, and how the babies grow and develop after they're born. Again, no experimental treatments are given; doctors just record the standard care and outcomes. This helps gather crucial information to better support women with Gaucher disease during pregnancy and ensure the best possible start for their babies.

Key takeaways

  • It helps doctors understand Gaucher disease better over time.
  • No new medicines or experimental treatments are given.
  • Information comes from your regular doctor's visits.
  • Special part for pregnant women with Gaucher disease.
  • Your data helps improve care for future patients.
  • You can stop participating at any time.

Who may be eligible?

Nearly anyone with a confirmed diagnosis of Gaucher disease can be part of the main study. This means you would have had tests that show a lack of a specific enzyme (beta-glucocerebrosidase) or a change in a particular gene associated with the disease. Your doctor would need to get your permission to share your health information for the study.

If you are a woman with Gaucher disease and are pregnant, or have been pregnant, you might also be able to join the special pregnancy part of the study. You would need to already be part of the main Gaucher Registry. You would also need to sign a consent form, giving your permission for your information to be collected.

There are no specific reasons that would stop someone from taking part in either the main study or the pregnancy study, as long as they meet the basic requirements mentioned above.

Quick self-check
  • Do I have a confirmed diagnosis of Gaucher disease?
  • Am I willing to allow my doctor to share my health information?
  • If pregnant or previously pregnant, am I currently in the main Gaucher Registry?
  • If pregnant or previously pregnant, am I willing to sign a consent form for the pregnancy sub-study?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

Taking part in this study is generally designed to fit into your existing medical care. You won't be given any experimental treatments; you'll continue to receive your usual care as decided by your doctor. Your participation means that your doctor will share information about your health, your condition, and any treatments you receive with the registry. This data comes from your routine clinic visits and assessments.

If you join the pregnancy sub-registry, information about your medical history, your pregnancy journey (including birth details), will be collected. If you agree, data about your baby's growth and health will also be collected up to when they are three years old. The total duration of your participation is ongoing, as the registry tracks health over many years, but your involvement is always voluntary.

Potential risks and benefits

The main benefit of taking part is contributing to a greater understanding of Gaucher disease, which can lead to better care for all patients in the future. There are no additional physical risks as you are not taking new medicines or having extra tests solely for this study; all care is determined by your doctor. Your personal information will be handled confidentially. You are always free to change your mind and withdraw from the study at any time without it affecting the care you receive from your doctor.

Locations (318)

  • Phoenix Children's Hospital- Site Number : 840003
    Phoenix, United States· Recruiting
  • University of Arizona- Site Number : 840015
    Tucson, United States· Recruiting
  • Arkansas Children's Hospital- Site Number : 840109
    Little Rock, United States· Recruiting
  • University of Arkansas for Medical Sciences- Site Number : 840113
    Little Rock, United States· Recruiting
  • Tower Hematology Oncology Medical Group- Site Number : 840056
    Beverly Hills, United States· Recruiting
  • University of California at Irvine- Site Number : 840036
    Irvine, United States· Recruiting
  • Southern California Permanente Medical Group- Site Number : 840108
    Los Angeles, United States· Recruiting
  • USC Health Sciences Center Dept of Genetics- Site Number : 840082
    Los Angeles, United States· Completed
  • Children's Hospital of Orange County- Site Number : 840074
    Orange, United States· Recruiting
  • UC Davis MIND Institute- Site Number : 840010
    Sacramento, United States· Recruiting
  • Kaiser Permanente Sacramento Medical Center- Site Number : 840042
    Sacramento, United States· Completed
  • University of California at San Diego- Site Number : 840007
    San Diego, United States· Completed

+306 more sites — see the official record for the full list.

Common questions

What is Gaucher disease?

Gaucher disease is a rare genetic condition where certain fatty substances build up in the body's cells and organs, which can cause various health problems.

Will I have to take new medicine or treatments?

No, you won't. This study is observational, meaning doctors only record information about the standard care you are already receiving; no experimental treatments are involved.

How long does participation last?

Participation is ongoing, as the registry collects information over many years to track long-term health, but you can withdraw at any time.

Is my personal information kept private?

Yes, your personal and medical information will be kept confidential and handled very carefully to protect your privacy.

What if I get pregnant while already in the study?

If you are a woman with Gaucher disease already in the main study and become pregnant, you could then choose to join the special pregnancy sub-registry, if it is still active.

How to find out more

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

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "International Collaborative Gaucher Group (ICGG) Gaucher Dis…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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