Fabry Disease Clinical Trials

A listing of 9 Fabry Disease clinical trials actively recruiting volunteers for paid trials and research studies in various therapeutic areas.

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There are currently 9 active clinical trials seeking participants for Fabry Disease research studies. The states with the highest number of trials for Fabry Disease participants are California, Virginia, New York and Georgia.

Featured Trial

Healthy Participants Needed (Colonoscopy + Cancer Screening)

Recruiting

Earn $325 - $475 in electronic payment card compensation for your time and effort by participating in a clinical study to develop a blood test that may one day help screen for colon cancer. Take a quick quiz to see if you qualify.

Conditions:

Healthy

Healthy Volunteers

Healthy Subjects

Healthy Volunteer

Healthy Participants

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Featured Trial

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Recruiting

Retatrutide (formerly M25) is an investigational peptide being studied for its potential in supporting weight management and metabolic health. It acts on GLP-1, GIP, and glucagon receptors to influence appetite, energy regulation, and fat metabolism.

Conditions:

Healthy

Learn More

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Conditions:

Overweight

Overweight and Obesity

Obesity

Weight Loss

Morbid Obesity

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Featured Trial

Birth Control Clinical Research Study

Recruiting

Living with obesity and looking for a birth control option that is right for you? Clinical trials are now enrolling women with a body mass index (BMI) of 30 or more in the area to help study a potential new progestin-only contraceptive patch. Participating in research studies can help advance birth control options. Don’t let financial stress hold you back. All study-related assessments, care, and study patch are available at no cost. You may also be reimbursed for study-related travel.

Conditions:

Obesity

Overweight

Overweight and Obesity

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Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

NCT01581424

Recruiting

The investigators will perform a study with two major components. The first is a natural history study of untreated Fabry patients. This study component will detail kidney microscopic structural changes in Fabry patients before starting enzyme replacement therapy and will correlate these changes with kidney function, including glomerular filtration rate and urinary albumin excretion rate. The investigators will perform studies on samples obtained at baseline, or before enzyme replacement therapy is initiated. The goal of our study is to find kidney microscopic changes in the biopsies that are associated with kidney disfunction. Our hypotheses for this study are: 1. Much of the natural history of Fabry renal structural changes will occur without detectable renal functional alterations. 2. Structural changes associated with the initial onset of proteinuria and those associated with the subsequent progressive loss of filtration function will differ and will be best described by non-linear models. 3. There will be sufficient precision of Fabry renal structural-functional relationships to support renal structure as an acceptable clinical trial surrogate endpoint for later renal functional deterioration. The second component examines the effects of age and gender at start of enzyme replacement therapy (ERT), as well as dosage levels of ERT on the renal cellular clearance of GL3 from Fabry patients by comparing baseline to follow-up kidney biopsies performed 5, 11, and 60 months later, with all comparisons matched for ERT treatment duration. Our hypotheses for this component of the study are as follows: 1. Enzyme Replacement Therapy(ERT) instituted at younger ages is more effective in reducing podocytes(PC),distal tubular cells(DTC),and arterial smooth muscle cells (ASMC)GL-3 than in older Fabry patients. 2. Earlier institution of ERT will stabilize PC numbers while later ERT institution, especially in proteinuric adults, may not prevent progressive decline in PC numbers and associated glomerular sclerosis, tubulointerstitial injury, and GFR loss. 3. Whereas lower ERT dose may effectively clear GL-3 from endothelial and mesangial cells, it will be less effective in clearing GL-3 from PC and also from DTC and ASMC. 4. Affected cells will be cleared of GL-3 equivalently in females and males. Read Less

Gender:

ALL

Ages:

Between 1 year and 75 years

Trial Updated:

08/21/2025

Locations: University of Minnesota, Minneapolis, Minnesota +1 locations

Conditions: Fabry Disease

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Fabry Disease Registry & Pregnancy Sub-registry

NCT00196742

Recruiting

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 F... Read More

Gender:

ALL

Ages:

All

Trial Updated:

08/07/2025

Locations: University of Alabama Birmingham- Nephrology- Site Number : 840018, Birmingham, Alabama +275 locations

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 Childrens 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

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

Children's Hospital Oakland- Site Number : 840029, Oakland, California

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

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

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

Kaiser Permanente Hospital- Site Number : 840042, Sacramento, California

University of California at San Diego- Site Number : 840007, San Diego, California

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

Yale - Site Number : 840047, New Haven, Connecticut

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

University Hematology Oncology- 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 Miami SOM- Site Number : 840006, Miami, Florida

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

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

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

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

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

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

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

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

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

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

Tulane University Medical Center- Site Number : 840001, New Orleans, Louisiana

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

John Hopkins- Site Number : 840044, Baltimore, Maryland

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

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

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

Baystate Health- Site Number : 840002, Springfield, Massachusetts

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

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

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

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

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

Children's Hospital and Clinics of Minnesota- Site Number : 840046, Minneapolis, Minnesota

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

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

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

Shodair Children's Hospital- Site Number : 840090, Helena, Montana

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

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

Cooper Health Center- Site Number : 840098, Camden, New Jersey

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Ohio State University Department of Genetics- Site Number : 840097, Columbus, Ohio

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

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

Oregon Health and Scinece University- Site Number : 840038, Portland, Oregon

Geisinger- Site Number : 840111, Danville, Pennsylvania

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Investigational Site Number : 153032, Ciudad Autónoma de Buenos Aires, Buenos Aires

Investigational Site Number : 153172, Buenos Aires, Not set

Investigational Site Number : 153130, Buenos Aires, Not set

Investigational Site Number : 153194, Catamarca, Not set

Investigational Site Number : 032005, La Rioja, Not set

Investigational Site Number : 032004, Santa Fe, Not set

Investigational Site Number : 036013, Westmead, New South Wales

Investigational Site Number : 151069, Westmead, New South Wales

Investigational Site Number : 153063, Brisbane, Queensland

Investigational Site Number : 151002, Adelaide, South Australia

Investigational Site Number : 153040, Parkville, Victoria

Investigational Site Number : 153090, Perth, Western Australia

Investigational Site Number : 150722, Antwerp, Not set

Investigational Site Number : 153133, Antwerp, Not set

Investigational Site Number : 153001, Brussels, Not set

Investigational Site Number : 0560008, Brussels, Not set

Investigational Site Number : 150517, Brussels, Not set

Investigational Site Number : 151668, Leuven, Not set

Investigational Site Number : 154275, Liège, Not set

Investigational Site Number : 153100, Seraing, Not set

Clínica Senhor do Bonfim LTDA- Site Number : 076008, Feira de Santana, Bahia

Medicordis- Site Number : 076009, Brasília, Distrito Federal

Hospital Geral e Maternidade de Cuiabá- Site Number : 076006, Cuiabá, Mato Grosso

CTDR - Centro de Tratamento de Doenças Renais EIRELI- Site Number : 076007, Uberlândia, Minas Gerais

Instituto do Rim do Paraná- Site Number : 076004, Curitiba, Paraná

Instituto de Medicina Integral Professor Fernando Figueira - IMIP - Site Number : 076005, Recife, Pernambuco

Saúde Center Clínica - Clínica Médica Nossa Senhora de Fátima - Site Number : 076002, Tapejara, Rio Grande Do Sul

Hospital Universitário Professor Polydoro Ernani de São Thiago - UFSC- Site Number : 076012, Florianopolis, Santa Catarina

Hospital e Maternidade Celso Pierro - PUC-Campinas- Site Number : 076013, Campinas, São Paulo

Serviço de Terapia Renal de Ourinhos- Site Number : 076011, Ourinhos, São Paulo

Instituto de Genética e Erros Inatos do Metabolismo- Site Number : 076001, São Paulo, Not set

Investigational Site Number : 153169, Sofia, Not set

Investigational Site Number : 100001, Sofia, Not set

Investigational Site Number : 124014, Calgary, Alberta

Investigational Site Number : 124002, Edmonton, Alberta

Investigational Site Number : 124010, Vancouver, British Columbia

Investigational Site Number : 124011, Vancouver, British Columbia

Investigational Site Number : 124019, Winnipeg, Manitoba

Investigational Site Number : 124022, Moncton, New Brunswick

Investigational Site Number : 124023, St. John's, Newfoundland and Labrador

Investigational Site Number : 124008, Halifax, Nova Scotia

Investigational Site Number : 124005, Kingston, Ontario

Investigational Site Number : 124021, London, Ontario

Investigational Site Number : 124006, Toronto, Ontario

Investigational Site Number : 124009, Toronto, Ontario

Investigational Site Number : 124013, Montreal, Quebec

Investigational Site Number : 124001, Sherbrooke, Quebec

Investigational Site Number : 152004, Antofagasta, Not set

Investigational Site Number : 152001, Coquimbo, Not set

Investigational Site Number : 152003, Iquique, Not set

Investigational Site Number : 152002, Santiago, Not set

Investigational Site Number : 152005, Vallenar, Not set

Investigational Site Number : 153165, Beijing, Not set

Investigational Site Number : 1560003, Hangzhou, Not set

Investigational Site Number : 1560002, Jinan, Not set

Investigational Site Number : 1560004, Nanjing, Not set

Investigational Site Number : 1560001, Shanghai, Not set

Investigational Site Number : 153163, Shanghai, Not set

Investigational Site Number : 170001, Bogotá, Not set

Investigational Site Number : 1910001, Rijena, Not set

Investigational Site Number : 1910002, Zagreb, Not set

Investigational Site Number : 153126, Praha 2, Not set

Investigational Site Number : 153041, Copenhagen, Not set

Investigational Site Number : 151229, København Ø, Not set

Investigational Site Number : 153106, Tartu, Not set

Investigational Site Number : 153024, Turku, Not set

Investigational Site Number : 250005, Bordeaux, Not set

Investigational Site Number : 250002, BRON Cedex, Not set

Investigational Site Number : 250001, Garches, Not set

Investigational Site Number : 250007, Lille, Not set

Investigational Site Number : 250004, St-priest-en-jarez, Not set

Investigational Site Number : 250009, Strasbourg, Not set

Investigational Site Number : 153013, Berlin, Not set

Investigational Site Number : 154150, Gießen, Not set

Investigational Site Number : 153147, Münster, Not set

Investigational Site Number : 153011, Würzburg, Not set

Investigational Site Number : 344001, Kowloon, Not set

Investigational Site Number : 3480002, Budapest, Not set

Investigational Site Number : 3480003, Létavértes, Not set

Investigational Site Number : 3480005, Pécs, Not set

Investigational Site Number : 3480004, Sopron, Not set

Investigational Site Number : 356003, Ahmedabad, Not set

Investigational Site Number : 356001, Kolkata, Not set

Investigational Site Number : 3560004, Secunderabad, Not set

Investigational Site Number : 356002, Vellore, Not set

Investigational Site Number : 360001, Jakarta, Not set

Investigational Site Number : 380002, Bassano Del Grappa (VI), Not set

Investigational Site Number : 380019, Bologna, Not set

Investigational Site Number : 380004, Firenze, Not set

Investigational Site Number : 380013, Firenze, Not set

Investigational Site Number : 380005, Genova, Not set

Investigational Site Number : 380009, Milano, Not set

Investigational Site Number : 380006, Milano, Not set

Investigational Site Number : 380003, Modena, Not set

Investigational Site Number : 380010, Monza (MB), Not set

Investigational Site Number : 380008, Napoli, Not set

Investigational Site Number : 380007, Rimini, Not set

Investigational Site Number : 380012, Roma, Not set

Investigational Site Number : 380011, Torino, Not set

Investigational Site Number : 380001, Udine, Not set

Investigational Site Number : 380014, Vittoria (RG), Not set

Investigational Site Number : 392007, Kobe, Hyogo

Investigational Site Number : 153201, Nangoku-shi, Kochi

Investigational Site Number : 153205, Abeno-ku, Osaka

Investigational Site Number : 153217, Chiba, Not set

Investigational Site Number : 392006, Niigata, Not set

Investigational Site Number : 153204, Tokyo, Not set

Investigational Site Number : 153178, Yangsan-si, Gyeongsangnam-do

Investigational Site Number : 154208, Yangsan, Gyeongsangnam-do

Investigational Site Number : 153180, Seoul, Seoul-teukbyeolsi

Investigational Site Number : 153196, Seoul, Seoul-teukbyeolsi

Investigational Site Number : 154246, Busan, Not set

Investigational Site Number : 153191, Daejeon, Not set

Investigational Site Number : 153228, Jeju-si, Not set

Investigational Site Number : 153192, Seoul, Not set

Investigational Site Number : 151149, Seoul, Not set

Investigational Site Number : 153188, Seoul, Not set

Investigational Site Number : 153212, Seoul, Not set

Investigational Site Number : 153222, Seoul, Not set

Investigational Site Number : 154273, Suwon-si, Not set

Investigational Site Number : 154244, Vilnius, Not set

Investigational Site Number : 154652, Vilnius, Not set

Investigational Site Number : 458001, Jalan Pahang, Not set

Investigational Site Number : 458002, Kuala Lumpur, Not set

Investigational Site Number : 151171, Amsterdam, Not set

Investigational Site Number : 153004, Amsterdam, Not set

Investigational Site Number : 153141, Bergen, Not set

Investigational Site Number : 604002, Callao, Not set

Investigational Site Number : 608001, Manila, Not set

Investigational Site Number : 150575, Warszawa, Not set

Investigational Site Number : 620005, Lisboa, Not set

Investigational Site Number : 620001, Porto, Not set

Investigational Site Number : 154099, Bucuresti, Not set

Investigational Site Number : 154164, Bucuresti, Not set

Investigational Site Number : 153081, Cluj-napoca, Not set

Investigational Site Number : 643001, Moscow, Not set

Investigational Site Number : 643003, Nizhny Novgorod, Not set

Investigational Site Number : 643002, Saint-Peterburg, Not set

Investigational Site Number : 682002, Al Mubarraz, Not set

Investigational Site Number : 6880001, Novi Sad, Not set

Investigational Site Number : 702001, Singapore, Not set

Investigational Site Number : 703001, Bratislava, Not set

Investigational Site Number : 705001, Slovenj Gradec, Not set

Investigational Site Number : 154146, A Coruña, A Coruña [La Coruña]

Investigational Site Number : 154159, A Coruña, A Coruña [La Coruña]

Investigational Site Number : 153237, Barcelona, Barcelona [Barcelona]

Investigational Site Number : 154147, Barcelona, Barcelona [Barcelona]

Investigational Site Number : 154204, Barcelona, Catalunya [Cataluña]

Investigational Site Number : 154135, Barakaldo, Pais Vasco

Investigational Site Number : 153213, Vigo, Pontevedra [Pontevedra]

Investigational Site Number : 154136, Tarragona, Tarragona [Tarragona]

Investigational Site Number : 153067, Barcelona, Not set

Investigational Site Number : 153077, Barcelona, Not set

Investigational Site Number : 154123, Barcelona, Not set

Investigational Site Number : 153050, Göteborg, Not set

Investigational Site Number : 151484, Stockholm, Not set

Investigational Site Number : 154093, Chiayi, Not set

Investigational Site Number : 152152, Hualien, Not set

Investigational Site Number : 153088, Kaohsiung City, Not set

Investigational Site Number : 152246, Kaohsiung City, Not set

Investigational Site Number : 152244, Kaohsiung, Not set

Investigational Site Number : 151534, New Taipei City, Not set

Investigational Site Number : 151500, Putz City, Chia-yi, Not set

Investigational Site Number : 151179, Taichung, Not set

Investigational Site Number : 151181, Taichung, Not set

Investigational Site Number : 153248, Tainan City, Not set

Investigational Site Number : 152153, Tainan City, Not set

Investigational Site Number : 151182, Taipei City, Not set

Investigational Site Number : 152226, Taipei City, Not set

Investigational Site Number : 152321, Taipei, Not set

Investigational Site Number : 153158, Taoyuan City, Not set

Investigational Site Number : 764003, Bangkok, Not set

Investigational Site Number : 764004, Bangkok, Not set

Investigational Site Number : 764001, Bangkok, Not set

Investigational Site Number : 764005, Khon Kaen, Not set

Investigational Site Number : 151340, Cambridge, Cambridgeshire

Investigational Site Number : 152287, Birmingham, England

Investigational Site Number : 151646, London, London, City Of

Investigational Site Number : 150303, London, London, City Of

Investigational Site Number : 152361, Salford, Manchester

Investigational Site Number : 154196, Belfast, Not set

Investigational Site Number : 151123, London, Not set

Investigational Site Number : 150455, Manchester, Not set

Investigational Site Number : 154212, Hanoi, Not set

Conditions: Fabry Disease

Learn More ›

A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

NCT04020055

Recruiting

An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)

Gender:

ALL

Ages:

18 years and above

Trial Updated:

07/24/2025

Locations: Emory University, Atlanta, Georgia +14 locations

Conditions: Fabry Disease

Learn More ›

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

NCT06270316

Recruiting

The main goals of this clinical study are to characterize safety and PK/PD of AMT-191 i.e. if drug doses used in the study are safe and tolerable and to understand how it acts in the body of people with Fabry disease.

Gender:

MALE

Ages:

Between 18 years and 50 years

Trial Updated:

07/24/2025

Locations: The Kirklin Clinic Of university of Alabama Birmingham Hospital, Birmingham, Alabama +7 locations

Conditions: Fabry Disease

Learn More ›

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066

Recruiting

This is a global prospective observational study of women with Fabry disease and their infants during pregnancy and/or breastfeeding. The study will evaluate outcomes of pregnancy and/or breastfeeding in women and infants exposed to migalastat.

Gender:

FEMALE

Ages:

All

Trial Updated:

07/02/2025

Locations: Amicus Therapeutics, Inc. Pregnancy Registry, Philadelphia, Pennsylvania

Conditions: Fabry Disease

Learn More ›

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

Recruiting

An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.

Gender:

ALL

Ages:

Between 2 years and 11 years

Trial Updated:

07/01/2025

Locations: Lysosomal and Rare Disorders Research and Treatment Center, Inc., Fairfax, Virginia

Conditions: Fabry Disease

Learn More ›

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

NCT06328608

Recruiting

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents with Fabry Disease.

Gender:

ALL

Ages:

Between 2 years and 17 years

Trial Updated:

12/18/2024

Locations: Phoenix Children's, Phoenix, Arizona +11 locations

Conditions: Fabry Disease

Learn More ›

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

NCT06663358

Recruiting

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

Gender:

ALL

Ages:

18 years and above

Trial Updated:

11/14/2024

Locations: Infusion Associates, Grand Rapids, Michigan

Conditions: Fabry Disease

Learn More ›

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

NCT06065605

Recruiting

The purpose of this research is to collect biological samples (urine) to develop assays for immune biomarkers to possibly in the future be able to screen subjects with Fabry disease and be able to understand better progression of nephropathy in Fabry disease and predict nephropathy in Fabry disease.

Gender:

ALL

Ages:

Between 18 years and 80 years

Trial Updated:

09/29/2023

Locations: Lysosomal & Rare Disorders Research & Treatment Center, Rockville, Maryland +1 locations

Conditions: Fabry Disease

Learn More ›

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