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Gaucher disease: open trials in United States

17 open trials have a site in United States. Registry data; only the trial team decides who can take part.

  • NCT00029965

    Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

    Research · National Human Genome Research Institute (NHGRI)

    National Institutes of Health Clinical Center, Bethesda

    Registry updated 2026-11-05

  • NCT07886879

    Parkinson's Disease in Type 1 Gaucher Disease

    Research · Icahn School of Medicine at Mount Sinai

    Icahn School of Medicine at Mount Sinai, New York

    Registry updated 2026-11-05

  • NCT02437396

    Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

    Research · University of Minnesota

    University of Minnesota, Minneapolis

    Registry updated 2026-11-04

  • NCT00001215

    Genetic Studies of Lysosomal Storage Disorders

    Research · National Human Genome Research Institute (NHGRI)

    National Institutes of Health Clinical Center, Bethesda

    Registry updated 2026-10-29

  • NCT00358943

    International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

    Research · Genzyme, a Sanofi Company

    University Of Michigan Pediatrics- Site Number : 840107, Ann Arbor · University Of Colorado At Denver Genetics- Site Number : 840068, Aurora · Baltimore · Tower Hematology Oncology Medical Group- Site Number : 840056, Beverly Hills · Massachusetts General Hospital-Genetics- Site Number : 840062, Boston · Boston Children's Hospital- Site Number : 840092, Boston · Cooper Health Center- Site Number : 840098, Camden · Atrium Health Carolinas Medical Center Hospital- Site Number : 840065, Charlotte · University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078, Charlottesville · Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840011, Chicago · Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013, Chicago · Rush University Medical Center Genetics- Site Number : 840079, Chicago · Cincinnati Children's Hospital Medical Center- Site Number : 840026, Cincinnati · Cleveland Clinic - Cleveland- Site Number : 840048, Cleveland · University Hospitals Cleveland Medical Center- Site Number : 840119, Cleveland · University Of Missouri Health System Department Of Genetics- Site Number : 840031, Columbia · Nationwide Children's Hospital- Site Number : 840091, Columbus · University of Miami- Site Number : 840006, Coral Gables · University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075, Coral Springs · Baylor Institute of Metabolic Disease- Site Number : 840058, Dallas · Emory University School Of Medicine- Human Genetics- Site Number : 840060, Decatur · Children's Hospital of Michigan- Site Number : 840066, Detroit · Lsd Data Registry Site Llc- Site Number : 840094, Dublin · Duke University Medical Center Genetics Dept- Site Number : 840037, Durham · O & O Alpan- Site Number : 840025, Fairfax · Cook Children's Medical Center- Site Number : 840024, Fort Worth · Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019, Grand Rapids · Infusion Associates - Grand Rapids- Site Number : 840050, Grand Rapids · Greenwood Genetic Center - Greenville- Site Number : 840055, Greenville · Joseph M. Sanzari Children'S Hospital- Site Number : 840101, Hackensack · Penn State Health Milton South Hershey Medical Center- Site Number : 840126, Hershey · NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040, Huntington · Indianapolis University School Of Medicine- Site Number : 840027, Indianapolis · University of Iowa- Site Number : 840032, Iowa City · University Of Florida Pediatrics Genetics- Site Number : 840121, Jacksonville · The Atwal Clinic- Site Number : 840112, Jacksonville · Children's Mercy Adelle Hall Campus- Site Number : 840063, Kansas City · Children's Specialty Center - Las Vegas- Site Number : 840008, Las Vegas · Arkansas Children's Hospital- Site Number : 840109, Little Rock · University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock · Southern California Permanente Medical Group- Site Number : 840108, Los Angeles · Usc Health Sciences Center Dept Of Genetics- Site Number : 840082, Los Angeles · University of Louisville - Health Sciences Center- Site Number : 840030, Louisville · Milwaukee · Children's Hospitals and Clinics of Minnesota- Site Number : 840046, Minneapolis · M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076, Minneapolis · Children's Hospitals and Clinics of Minnesota- Site Number : 840114, Minneapolis · Atlantic Health System- Site Number : 840099, Morristown · Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049, Nashville · Saint Peter's University Hospital - New Brunswick- Site Number : 840016, New Brunswick · Yale University School of Medicine- Site Number : 840047, New Haven · Northwell Health- Site Number : 840102, New Hyde Park · Tulane University School of Medicine- Site Number : 840001, New Orleans · Icahn School of Medicine at Mount Sinai- Site Number : 840005, New York · Metropolitan Hospital Center- Site Number : 840110, New York · Columbia University Irving Medical Center- Site Number : 840125, New York · Children's Hospital of The King's Daughters- Site Number : 840072, Norfolk · University of Nebraska Medical Center- Site Number : 840084, Omaha · Children's Hospital of Orange County- Site Number : 840074, Orange · University of California Irvine Medical Center- Site Number : 840036, Orange · St. Joseph's Regional Medical Center - Paterson- Site Number : 840057, Paterson · Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089, Philadelphia · The Children's Hospital of Philadelphia- Site Number : 840034, Philadelphia · Phoenix Children's Hospital- Site Number : 840003, Phoenix · UPMC Children's Hospital of Pittsburgh- Site Number : 840023, Pittsburgh · Maine Medical Partners Pediatric Specialty Care- Site Number : 840064, Portland · Oregon Health & Science University (OHSU)- Site Number : 840080, Portland · Rhode Island Hospital Genetics- Site Number : 840053, Providence · University of Rochester Medical Center- Site Number : 840105, Rochester · Uc Davis Mind Institute- Site Number : 840010, Sacramento · Kaiser Permanente Sacramento Medical Center- Site Number : 840042, Sacramento · University of Utah- Site Number : 840043, Salt Lake City · University of California at San Diego- Site Number : 840007, San Diego · University of California San Francisco - Parnassus Campus- Site Number : 840051, San Francisco · Seattle Children's Hospital- Site Number : 840028, Seattle · University of Washington Medical Center- Site Number : 840059, Seattle · Stanford University Medical Center- Site Number : 840022, Stanford · Washington University- Site Number : 840100, St Louis · SUNY Upstate Medical University - Site Number : 840052, Syracuse · Mary Bridge Children's Hospital & Health Center- Site Number : 840115, Tacoma · University Of Florida-Genetics- Site Number : 840096, Tampa · Tampa · University of Arizona- Site Number : 840015, Tucson · New York Medical College- Site Number : 840039, Valhalla · Children's National Medical Center - Washington- Site Number : 840067, Washington D.C. · Washington D.C.

    Registry updated 2026-10-14

  • NCT07223944

    A Gaucher Disease Gene Therapy Trial With FLT201

    Trial Treatment · Phase 3 · Spur Therapeutics

    Emory Healthcare, Atlanta · University of Colorado Anschutz, Aurora · Cincinnati Children's Hospital Medical Center, Cincinnati · University of Texas, Southwestern, Dallas · Duke University Medical Center, Durham · Lysosomal Rare Disorders Research and Treatment Center, Fairfax · Hackensack University Medical Center, Hackensack · University of Iowa Health Care, Iowa City · Kaiser Permanente - CA, Los Angeles · UCLA Health, Los Angeles · University of Minnesota, Minneapolis · NYU Langone Health, New York · Children's Hospital of Philadelphia, Philadelphia · UPMC Children's Hospital of Pittsburgh, Pittsburgh · University of California, San Francisco (UCSF), San Francisco

    Registry updated 2026-10-14

  • NCT03291223

    Gaucher Disease Outcome Survey (GOS)

    Research · Shire

    Lexington

    Registry updated 2026-09-15

  • NCT03190837

    A Long-term Follow-up Study of Gaucher Disease

    Research · Duke University

    Duke University Medical Center, Durham

    Registry updated 2026-09-03

  • NCT07758816

    Advancing Integrated Therapies for Gaucher Disease

    Research · Yale University

    Yale-New Haven Health System, New Haven

    Registry updated 2026-08-11

  • NCT05586243

    MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

    Research · University of Minnesota

    University of Minnesota, Minneapolis

    Registry updated 2026-08-04

  • NCT05368038

    ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

    Research · Albert Einstein College of Medicine

    Maimonides Medical Center, Brooklyn · NYU Langone Hospital - Brooklyn, Brooklyn · North Shore University Hospital, Manhasset · NYU Langone Health - Tisch Hospital, New York · Mount Sinai West, New York · Mount Sinai Hospital, New York · Long Island Jewish Medical Center, Queens · Jack D. Weiler Hospital, The Bronx · ScreenPlus Coordinating Core, Children's Hospital at Montefiore, The Bronx

    Registry updated 2026-07-28

  • NCT05619900

    Registry of Patients Diagnosed With Lysosomal Storage Diseases

    Research · University of California, San Francisco

    University of California San Francisco, San Francisco

    Registry updated 2026-04-08

  • NCT05843552

    Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

    Research · University of Minnesota

    University of Minnesota, Minneapolis

    Registry updated 2026-04-06

  • NCT04532047

    PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

    Trial Treatment · Phase 1 · University of California, San Francisco

    University of California, San Francisco

    Registry updated 2026-03-17

  • NCT03333200

    Longitudinal Study of Neurodegenerative Disorders

    Research · University of Pittsburgh

    UPMC Children's Hospital of Pittsburgh, Pittsburgh

    Registry updated 2026-02-09

  • NCT04528355

    Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

    Research · Paul Szabolcs

    UPMC Children's Hospital of Pittsburgh, Pittsburgh

    Registry updated 2026-01-13

  • NCT01962415

    Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

    Trial Treatment · Phase 2 · Paul Szabolcs

    UPMC Children's Hospital of Pittsburgh, Pittsburgh

    Registry updated 2025-12-15

All Gaucher disease trials and the map

This page is not medical advice. Talk to your doctor before contacting a trial.

Gaucher disease trials in United States – Frontlight Research