Fabry Disease Registry & Pregnancy Sub-registry (NCT00196742)
Fabry Disease Registry Protocol
🩺 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 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.
Who can join: Review the eligibility criteria below.
Location: University of Alabama Birmingham- Nephrology- Site Number : 840018 — Birmingham, Alabama
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (284 Sites)
Showing the first 25 of 284 sites.
- University of Alabama Birmingham- Nephrology- Site Number : 840018 — Birmingham, Alabama
- University of Alabama Birmingham- Nephrology- Site Number : 840073 — Birmingham, Alabama
- Phoenix Children's Hospital- Site Number : 840003 — Phoenix, Arizona
- University of Arizona- Site Number : 840015 — Tucson, Arizona
- Arkansas Children's Hospital- Site Number : 840109 — Little Rock, Arkansas
- University of Arkansas for Medical Sciences- Site Number : 840113 — Little Rock, Arkansas
- University of California at Irvine- Site Number : 840036 — Irvine, California
- Southern California Permanente Medical Group- Site Number : 840108 — Los Angeles, California
- USC Health Sciences Center Dept of Genetics- Site Number : 840082 — Los Angeles, California
- University Of California Los Angeles- Site Number : 840088 — Los Angeles, California
- UCSF Benioff Children's Hospital - Oakland- Site Number : 840029 — Oakland, California
- Children's Hospital of Orange County- Site Number : 840074 — Orange, California
- UC Davis MIND Institute- Site Number : 840010 — Sacramento, California
- Kaiser Permanente Sacramento Medical Center- Site Number : 840042 — Sacramento, California
- University of California at San Diego- Site Number : 840007 — San Diego, California
- University of California San Francisco - Parnassus Campus- Site Number : 840051 — San Francisco, California
- Stanford University Medical Center- Site Number : 840022 — Stanford, California
- Yale University School of Medicine- Site Number : 840047 — New Haven, Connecticut
- Children's National Medical Center - Washington- Site Number : 840067 — Washington D.C., District of Columbia
- University of Miami- Site Number : 840006 — Coral Gables, Florida
- University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075 — Coral Springs, Florida
- University of Florida Dept of Genetics- Site Number : 840083 — Gainesville, Florida
- University of Florida Pediatrics Genetics- Site Number : 840121 — Jacksonville, Florida
- University of Florida-Genetics- Site Number : 840096 — Tampa, Florida
- Emory University School of Medicine- Human Genetics- Site Number : 840060 — Decatur, Georgia
📞 Contact the Study Coordinator
Phone: (800) 633-1610
Email: contact-us@sanofi.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 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 § 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.
More studies in Fabry Disease
- A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease — Birmingham, Alabama
- A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease — Birmingham, Alabama
- Long-Term Follow-up of Subjects Who Were Treated With ST-920 — Irvine, California
- An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease — La Jolla, California
- A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease — Birmingham, Alabama
- A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease — Atlanta, Georgia
Explore by condition: All Fabry Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.