Fabry Disease Registry & Pregnancy Sub-registry

Description

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry 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 primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. 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

Fabry Disease

Study Overview

Study Details

Study overview

The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry 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 primary objectives of the Registry are: * To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; * To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; * To characterize and describe the Fabry population as a whole; * To evaluate the long-term safety and effectiveness of Fabrazyme® Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and irrespective of the commercial product with which she may be treated. Data from the Sub-registry are also used to fulfill various global regulatory requirements, to support product development/reimbursement, and for other research and non-research-related purposes. 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.

Fabry Disease Registry Protocol

Fabry Disease Registry & Pregnancy Sub-registry

Condition
Fabry Disease
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama Birmingham- Nephrology- Site Number : 840018, Birmingham, Alabama, United States, 35294

Birmingham

University of Alabama Birmingham- Nephrology- Site Number : 840073, Birmingham, Alabama, United States, 35294

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

Irvine

University of California at Irvine- Site Number : 840036, Irvine, California, United States, 92697

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

Los Angeles

UCLA School Of Medicine- Site Number : 840088, Los Angeles, California, United States, 90095

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

    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