Fabry Disease Registry & Pregnancy Sub-registry (NCT00196742)
๐ฉบ Plain-English Summary (8th-Grade Level)
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 Fabrazymeยฎ 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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (6 items) and avoid the listed exclusions (1 items).
Location: University of Alabama Birmingham- Nephrology- Site Number : 840018 โ Birmingham, Alabama
๐ "Do I Qualify?" โ 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- Exclusion Criteria Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.
๐ Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
๐ All Participating Trial Locations (284 Sites)
- University of Alabama Birmingham- Nephrology- Site Number : 840018 โ Birmingham, Alabama
- University of Alabama Birmingham- Nephrology- Site Number : 840073 โ Birmingham, Alabama
- Phoenix Children's Hospital- Site Number : 840003 โ Phoenix, Arizona
- University of Arizona- Site Number : 840015 โ Tucson, Arizona
- Arkansas Children's Hospital- Site Number : 840109 โ Little Rock, Arkansas
- University of Arkansas for Medical Sciences- Site Number : 840113 โ Little Rock, Arkansas
- University of California at Irvine- Site Number : 840036 โ Irvine, California
- Southern California Permanente Medical Group- Site Number : 840108 โ Los Angeles, California
- USC Health Sciences Center Dept of Genetics- Site Number : 840082 โ Los Angeles, California
- University Of California Los Angeles- Site Number : 840088 โ Los Angeles, California
- UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 โ Oakland, California
- Children's Hospital of Orange County- Site Number : 840074 โ Orange, California
- UC Davis MIND Institute- Site Number : 840010 โ Sacramento, California
- Kaiser Permanente Sacramento Medical Center- Site Number : 840042 โ Sacramento, California
- University of California at San Diego- Site Number : 840007 โ San Diego, California
- University of California San Francisco - Parnassus Campus- Site Number : 840051 โ San Francisco, California
- Stanford University Medical Center- Site Number : 840022 โ Stanford, California
- Yale University School of Medicine- Site Number : 840047 โ New Haven, Connecticut
- Children's National Medical Center - Washington- Site Number : 840067 โ Washington D.C., District of Columbia
- University of Miami- Site Number : 840006 โ Coral Gables, Florida
- University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075 โ Coral Springs, Florida
- University of Florida Dept of Genetics- Site Number : 840083 โ Gainesville, Florida
- University of Florida Pediatrics Genetics- Site Number : 840121 โ Jacksonville, Florida
- University of Florida-Genetics- Site Number : 840096 โ Tampa, Florida
- Emory University School of Medicine- Human Genetics- Site Number : 840060 โ Decatur, Georgia
๐ฐ Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA ยง 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.