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

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. 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.

Conditions

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

Study Overview

Study Details

Study overview

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.

Gaucher Disease Registry Protocol

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

Condition
Gaucher Disease
Intervention / Treatment

-

Contacts and Locations

Phoenix

Phoenix Children's Hospital- Site Number : 840003, Phoenix, Arizona, United States, 85013

Tucson

University of Arizona- Site Number : 840015, Tucson, Arizona, United States, 85724

Little Rock

Arkansas Childrens Hospital- Site Number : 840109, Little Rock, Arkansas, United States, 72202

Little Rock

University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock, Arkansas, United States, 72205

Beverly Hills

Tower Hematology/Oncology Medical Group- Site Number : 840056, Beverly Hills, California, United States, 90211

Irvine

University of California at Irvine- Site Number : 840036, Irvine, California, United States, 92617

Los Angeles

Southern California Permanente Medical Group- Site Number : 840108, Los Angeles, California, United States, 90027

Los Angeles

USC Health Sciences Center Dept of Genetics- Site Number : 840082, Los Angeles, California, United States, 90033

Orange

Children's Hospital of Orange County- Site Number : 840074, Orange, California, United States, 92868

Palo Alto

Stanford Unviersity MC Dept of Genetics- Site Number : 840022, Palo Alto, California, United States, 94305

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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.
  • * 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.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Genzyme, a Sanofi Company,

Study Director, STUDY_DIRECTOR, Genzyme, a Sanofi Company

Study Record Dates

2034-01-31