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

Conditions

• Cerebroside Lipidosis Syndrome
• Gaucher Disease
• Glucocerebrosidase Deficiency Disease
• Glucosylceramide Beta-Glucosidase Deficiency Disease

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Apr 1, 1991

Status verified

Apr 2026

Primary completion

Jan 31, 2034

Completion

Jan 31, 2034

Study Design

Enrollment

12,000 participants (estimated)

Arms

• Arm: Patients in ICGG Gaucher Registry
  No experimental intervention is given. A patient with Gaucher Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
• Arm: Pregnant women with confirmed diagnosis of Gaucher disease
  No experimental intervention is given. Pregnant women with confirmed diagnosis of Gaucher disease who are participating in the ICGG Gaucher Registry and consented to participate in the Gaucher Pregnancy Sub-registry, regardless of whether she is receiving disease-specific therapy and irrespective of the commercial product with which she may be treated.

Primary Outcome Measure

ICGG Gaucher Registry: To provide the Gaucher medical community with recommendations for monitoring patients and to provide reports on patient outcomes to help optimize patient care [ Time Frame: 42 years ]

Central Contacts

• Trial Transparency email recommended (Toll free number for US & Canada)
  800-633-1610
  [email protected]
• ICGG Gaucher Registry HelpLine
  +1(617) 252-7500

Locations (86)

Find similar trials in Phoenix, AZ

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