Tracks outcomes of people with Gaucher disease, including during pregnancy
Follows people with Gaucher disease over time, with and without treatment, including pregnancy.
Plain-language title generated from the registry record
Registry title: International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Key facts
- Where
- Birmingham, Cambridge, London, Manchester, and Salford, United KingdomSee the 8 sites
Where in United Kingdom
- BirminghamBirmingham, United KingdomTaking people
- BirminghamBirmingham, United KingdomTaking people
- CambridgeCambridge, United KingdomTaking people
- LondonLondon, United KingdomTaking people
- LondonLondon, United KingdomTaking people
- LondonLondon, United KingdomTaking people
- ManchesterManchester, United KingdomTaking people
- SalfordSalford, United KingdomTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Genzyme, a Sanofi Company
- Trial code
- NCT00358943
- Record updated
- 14 October 2026
- Original description
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.Read full original descriptionShow less
Gaucher Disease Registry Protocol
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.
The objectives of the Registry are:
* To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and assessing therapeutic intervention. * To assist the Gaucher medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care. * To characterize the Gaucher disease population. * To evaluate the long-term effectiveness of imiglucerase and of eliglustat.
Gaucher Pregnancy Sub-registry: The primary objective of this Sub-registry is to track pregnancy outcomes, including complications and infant growth, in all women with Gaucher disease during pregnancy, regardless of whether they receive disease-specific therapy. No experimental intervention is given; thus a patient will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.If a patient consents to this Sub-registry, information about the patient's medical and obstetric history, pregnancy, and birth will be collected, and, if a patient consents to data collection for her infant, data on infant growth through month 36 postpartum will be collected.
Inclusion: ICGG Gaucher Registry; All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion in the Registry. Confirmed diagnosis is defined as a documented β-glucocerebrosidase deficiency and/or mutation in the β-glucocerebrosidase gene; For all patients, appropriate patient authorization will be obtained; Gaucher Pregnancy Sub-registry:; be enrolled in the ICGG Gaucher Registry; be pregnant, or have been pregnant with appropriate medical documentation available; provide a signed informed consent and authorization form(s) to participate in the Sub-Registry prior to any Sub-Registry-related data collection being performed.
Exclusion: No exclusion criteria for participation in the ICGG Gaucher Registry and Sub-registry.