Fabry Disease Registry & Pregnancy Sub-registry

Purpose

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.

Condition

  • Fabry Disease

Eligibility

Eligible Ages
All ages
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Fabry Registry: All patients with a confirmed diagnosis of Fabry disease who have signed the informed consent and patient authorization form(s) are eligible for inclusion. Confirmed diagnosis is defined as a documented deficiency in plasma or leukocyte αGAL (alpha-galactosidase) enzyme activity and/or mutation(s) in the gene coding for αGAL. - Fabry Pregnancy Sub-registry: - Eligible women must: - be enrolled in the Fabry 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.

Exclusion Criteria

Fabry Registry: There are no exclusion criteria. Fabry Pregnancy Sub-registry: There are no exclusion criteria.

Study Design

Phase
Study Type
Observational [Patient Registry]
Observational Model
Cohort
Time Perspective
Other

Arm Groups

ArmDescriptionAssigned Intervention
Patients with Fabry disease No experimental intervention is given. A patient with Fabry Disease will undergo clinical assessments and receive standard of care treatment as determined by the patient's physician.
Pregnant women with confirmed diagnosis of Fabry No experimental intervention is given. Pregnant women with confirmed diagnosis of Fabry that are participating in the Fabry Registry and consented to participate in the Fabry Sub-registry, regardless of whether she is receiving disease-specific therapy (such as ERT with agalsidase beta) and irrespective of the commercial product with which she may be treated.

Recruiting Locations

University of Alabama Birmingham- Nephrology- Site Number : 840073
Birmingham 4049979, Alabama 4829764 35294

Phoenix Children's Hospital- Site Number : 840003
Phoenix 5308655, Arizona 5551752 85013

University of Arizona- Site Number : 840015
Tucson 5318313, Arizona 5551752 85724

Arkansas Childrens Hospital- Site Number : 840109
Little Rock 4119403, Arkansas 4099753 72202

University of Arkansas for Medical Sciences- Site Number : 840113
Little Rock 4119403, Arkansas 4099753 72205

University of California at Irvine- Site Number : 840036
Irvine 5359777, California 5332921 92697

Southern California Permanente Medical Group- Site Number : 840108
Los Angeles 5368361, California 5332921 90027

UCLA School Of Medicine- Site Number : 840088
Los Angeles 5368361, California 5332921 90095

Children's Hospital of Orange County- Site Number : 840074
Orange 5379513, California 5332921 92868

Stanford Unviersity MC Dept of Genetics- Site Number : 840022
Palo Alto 5380748, California 5332921 94305

UC Davis MIND Institute- Site Number : 840010
Sacramento 5389489, California 5332921 95817

University of California at San Francisco- Site Number : 840051
San Francisco 5391959, California 5332921

Yale - Site Number : 840047
New Haven 4839366, Connecticut 4831725 06520

Children's National Medical Center- Site Number : 840067
Washington D.C. 4140963, District of Columbia 4138106 20010

University Hematology Oncology- Site Number : 840075
Coral Springs 4151909, Florida 4155751 33065

University of Florida Dept of Genetics- Site Number : 840083
Gainesville 4156404, Florida 4155751 32610

University of Florida Pediatrics Genetics- Site Number : 840121
Jacksonville 4160021, Florida 4155751 32207

University Of Miami SOM- Site Number : 840006
Miami 4164138, Florida 4155751 33136

University of Florida-Genetics- Site Number : 840096
Tampa 4174757, Florida 4155751 33606

Emory University School of Medicine- Human Genetics- Site Number : 840060
Decatur 4191124, Georgia 4197000 30033

University of Colorado at Denver Genetics- Site Number : 840068
Aurora 4883817, Illinois 4896861 80045

Ann and Robert Lurie Children's Hospital- Site Number : 840013
Chicago 4887398, Illinois 4896861 60611

Rush University Medical Center Genetics- Site Number : 840079
Chicago 4887398, Illinois 4896861 60612

Indianapolis University School of Medicine- Site Number : 840027
Indianapolis 4259418, Indiana 4921868 46202

University of Iowa- Site Number : 840032
Iowa City 4862034, Iowa 4862182 52242

University of Kansas Medical Center- Site Number : 840071
Kansas City 4273837, Kansas 4273857 66160

Ochsner Medical Center- Site Number : 840120
Baton Rouge 4315588, Louisiana 4331987 70809

Maine Medical Center Pediatrics- Site Number : 840064
Portland 4975802, Maine 4971068 04102

Massachusetts General Hospital-Genetics- Site Number : 840062
Boston 4930956, Massachusetts 6254926 02114

Brigham and Women's Hospital-Neurology- Site Number : 840103
Boston 4930956, Massachusetts 6254926 02115

Boston Children's Hospital- Site Number : 840092
Boston 4930956, Massachusetts 6254926 2115

Baystate Health- Site Number : 840002
Springfield 4951788, Massachusetts 6254926 01199

University of Michigan Pediatrics- Site Number : 840107
Ann Arbor 4984247, Michigan 5001836 48109

Children's Hospital of Michigan- Site Number : 840066
Detroit 4990729, Michigan 5001836 48201

Infusion Associates- Site Number : 840050
Grand Rapids 4994358, Michigan 5001836 49525

Children's Health Care- Site Number : 840114
Minneapolis 5037649, Minnesota 5037779 55404

University of Minnesota Medical Center Pediatrics- Site Number : 840076
Minneapolis 5037649, Minnesota 5037779 55455

University of Missouri Health System Department of Genetics- Site Number : 840031
Columibia, Missouri 4398678 65212

Washington University of St. Louis- Site Number : 840100
St Louis 4407066, Missouri 4398678 63110

University of Nebraska Medical Center- Pediatrics- Site Number : 840084
Omaha 5074472, Nebraska 5073708 68198

Children's Specialty Center of Nevada- Site Number : 840008
Las Vegas 5506956, Nevada 5509151 89109

Joseph M. Sanzari Children's Hospital- Site Number : 840101
Hackensack 5098706, New Jersey 5101760 07601

Atlantic Health System- Site Number : 840099
Morristown 5101427, New Jersey 5101760 07962

St. Peter's University Hospital- Site Number : 840016
New Brunswick 5101717, New Jersey 5101760 08901

St. Joseph's Children's Hospital- Site Number : 840057
Paterson 5102466, New Jersey 5101760 07503

Northwell Health- Site Number : 840102
Manhasset 5125766, New York 5128638 11030

New York University School Of Medicine- Site Number : 840040
New York 5128581, New York 5128638 10016

Metropolitan Hospital Center- Site Number : 840110
New York 5128581, New York 5128638 10029-7494

Icahn School of Medicine at Mount Sinai- Site Number : 840080
New York 5128581, New York 5128638 10029

State University of New York- Site Number : 840052
Syracuse 5140405, New York 5128638 13210

New York Medical College- Site Number : 840039
Valhalla 5142090, New York 5128638 10595

Carolinas Medical Center- Site Number : 840065
Charlotte 4460243, North Carolina 4482348 28232

Duke University Medical Center Genetics Dept- Site Number : 840045
Durham 4464368, North Carolina 4482348 27710

Cincinnati Children's Hospital-Genetics- Site Number : 840033
Cincinnati 4508722, Ohio 5165418

University Hospitals Cleveland Medical Center- Site Number : 840119
Cleveland 5150529, Ohio 5165418 44106

Cleveland Clinic Foundation Pediatrics- Site Number : 840048
Cleveland 5150529, Ohio 5165418 44195

Nationwide Children's Hospital- Site Number : 840091
Columbus 4509177, Ohio 5165418 43205-2664

LSD Data Registry Site LLC- Site Number : 840094
Dublin 5152333, Ohio 5165418 43017

Oregon Health and Science University- Site Number : 840116
Portland 5746545, Oregon 5744337 97239

Geisinger- Site Number : 840111
Danville 5186327, Pennsylvania 6254927 17822

Penn State University Medical Center-Neurology- Site Number : 840104
Hershey 5193342, Pennsylvania 6254927 17033

Children's Hospital of Philadelphia HUP Medical Genetics- Site Number : 840089
Philadelphia 4560349, Pennsylvania 6254927 19104

Children's Hospital of Philadelphia- Site Number : 840034
Philadelphia 4560349, Pennsylvania 6254927

University of Pittsburgh- Site Number : 840023
Pittsburgh 5206379, Pennsylvania 6254927 15224

Greenwood Genetcs- Site Number : 840055
Greenville 4580543, South Carolina 4597040 29605

Vanderbilt University Hospital-Pediatrics- Site Number : 840049
Nashville 4644585, Tennessee 4662168 38232

Baylor Research Center- Site Number : 840058
Dallas 4684888, Texas 4736286 75226

Cook Children's Health Care System- Site Number : 840024
Fort Worth 4691930, Texas 4736286 76104

University of Utah Department of Medical Genetics- Site Number : 840043
Salt Lake City 5780993, Utah 5549030 84132

O&O Alpan, LLC- Site Number : 840025
Fairfax 4758023, Virginia 6254928 22030

Seattle Children's Hospital- Site Number : 840028
Seattle 5809844, Washington 5815135 98105

University Of Washington Medical Center- Site Number : 840059
Seattle 5809844, Washington 5815135 98195

Multicare Health System ¿ Mary Bridge Childrens Hospital- Site Number : 840115
Tacoma 5812944, Washington 5815135 98415

Children's Hospital of Wisconsin-Pediatrics- Site Number : 840054
Milwaukee 5263045, Wisconsin 5279468 53226

More Details

NCT ID
NCT00196742
Status
Recruiting
Sponsor
Genzyme, a Sanofi Company

Study Contact

Trial Transparency
800-633-1610
contact-us@sanofi.com