UAMS - Translational Research Institute

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 Genders: 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

Arm	Description	Assigned 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, Alabama 35294

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

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

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

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

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

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

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

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

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

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

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

Loma Linda University Children's Hospital- Site Number : 840117
San Bernardino, California 92408

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

Yale - Site Number : 840047
New Haven, Connecticut 06520

Children's National Medical Center- Site Number : 840067
Washington, District of Columbia 20010

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

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

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

The Atwal Clinic- Site Number : 840112
Jacksonville, Florida 32216

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

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

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

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

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

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

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

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

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

University of Louisville- Genetics- Site Number : 840030
Louisville, Kentucky 40202

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

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

John Hopkins- Site Number : 840044
Baltimore, Maryland

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

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

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

Baystate Health- Site Number : 840002
Springfield, Massachusetts 01199

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

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

Spectrum for Health- Site Number : 840019
Grand Rapids, Michigan 49503

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

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

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

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

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

Billings Clinic - Billings- Site Number : 840118
Billings, Montana 59101

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

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

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

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

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

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

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

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

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

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

University of Rochester Medical Center SOM- Site Number : 840105
Rochester, New York 14642

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

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

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

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

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

UHCMC- Site Number : 840119
Cleveland, Ohio 44106

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

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

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

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

Geisinger- Site Number : 840111
Danville, Pennsylvania 17822

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

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

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

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

Rhode Island Hospital Genetics- Site Number : 840053
Providence, Rhode Island 02903

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

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

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

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

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

University of Virginia School of Medicine-Pediatrics- Site Number : 840078
Charlottesville, Virginia 22908

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

Children's Hospital of the Kings Daughters- Site Number : 840072
Norfolk, Virginia 23507

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

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

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

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

More Details

NCT ID: NCT00196742
Status: Recruiting
Sponsor: Genzyme, a Sanofi Company

Study Contact

Trial Transparency
800-633-1610
Contact-Us@sanofi.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.