Gaucher disease: trials for referral
35 open trials in the registries we read. Each row links to its registry record.
Registry data only. Frontlight does not judge whether a patient can take part: the trial team decides.
Where the open trials are
Type a city to sort by distance. The distance is worked out in your browser; the city is not sent anywhere.
35 trials shown
No phase1 site
RecruitingUnited States
NCT07886879
Parkinson's Disease in Type 1 Gaucher Disease
Icahn School of Medicine at Mount Sinai
Registry updated 5 Nov 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT00029965
Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
National Human Genome Research Institute (NHGRI)
Registry updated 5 Nov 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT02437396
Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
University of Minnesota
Registry updated 4 Nov 2026
Registry record: ClinicalTrials.govNo phase1 site
By invitation onlyUnited States
NCT00001215
Genetic Studies of Lysosomal Storage Disorders
National Human Genome Research Institute (NHGRI)
Registry updated 29 Oct 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited Kingdom
NCT06151002
PD Frontline (Part of RAPSODI GD) Remote Assessment of People With Parkinson's
University College, London
Registry updated 29 Oct 2026
Registry record: ClinicalTrials.govPhase 41 site
Not yet recruitingNetherlands
2026-527117-33-00
ONCE: Once daily eliglustat in stable CYP2D6 intermediate and extensive metabolizers with Gaucher disease type 1
Amsterdam UMC Stichting
Registry updated 22 Oct 2026
Registry record: EU CTISPhase 2/30 sites
Not yet recruiting
NCT07885917
Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal Disorders
Azafaros B.V.
Registry updated 21 Oct 2026
Registry record: ClinicalTrials.govPhase 332 sites
RecruitingArgentina, Australia, Brazil, Canada, Spain, United Kingdom
NCT07223944
A Gaucher Disease Gene Therapy Trial With FLT201
Spur Therapeutics
Registry updated 14 Oct 2026
Registry record: ClinicalTrials.govNo phase317 sites
RecruitingUnited Arab Emirates, Argentina, Australia, Belgium, Bulgaria, Brazil
NCT00358943
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
Genzyme, a Sanofi Company
Registry updated 14 Oct 2026
Registry record: ClinicalTrials.govNo phase0 sites
Not yet recruiting
NCT05669729
A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion
Takeda
Registry updated 1 Oct 2026
Registry record: ClinicalTrials.govPhase 31 site
Not yet recruitingChina
NCT07847411
Study of Velaglucerase Beta ERT in Children With Gaucher Disease
CANbridge Life Sciences Ltd.
Registry updated 29 Sept 2026
Registry record: ClinicalTrials.govNo phase0 sites
Not yet recruiting
NCT07845188
Nutritional Status of Gaucher Disease Patients
Assiut University
Registry updated 28 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT03291223
Gaucher Disease Outcome Survey (GOS)
Shire
Registry updated 15 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT03190837
A Long-term Follow-up Study of Gaucher Disease
Duke University
Registry updated 3 Sept 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT07758816
Advancing Integrated Therapies for Gaucher Disease
Yale University
Registry updated 11 Aug 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05586243
MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
University of Minnesota
Registry updated 4 Aug 2026
Registry record: ClinicalTrials.govNo phase9 sites
By invitation onlyUnited States
NCT05368038
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Albert Einstein College of Medicine
Registry updated 28 Jul 2026
Registry record: ClinicalTrials.govPhase 27 sites
Not yet recruitingIndia, Pakistan
NCT07715084
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
Azafaros B.V.
Registry updated 20 Jul 2026
Registry record: ClinicalTrials.govPhase 1/20 sites
Not yet recruiting
NCT07603050
A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease
Shanghai Vitalgen BioPharma Co., Ltd.
Registry updated 22 May 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingIsrael
NCT05253560
Prodromal Parkinsonian Features in GBA1 Mutation Carriers
Shaare Zedek Medical Center
Registry updated 29 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingIsrael
NCT04388969
World Data on Ambroxol for Patients With GD and GBA Related PD
Shaare Zedek Medical Center
Registry updated 29 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05619900
Registry of Patients Diagnosed With Lysosomal Storage Diseases
University of California, San Francisco
Registry updated 8 Apr 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT05843552
Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease
University of Minnesota
Pre-treatment window Registry sentence: “Restricted to participants who are untreated, obligate carriers and healthy controls.”
Registry updated 6 Apr 2026
Registry record: ClinicalTrials.govPhase 11 site
RecruitingUnited States
NCT04532047
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
University of California, San Francisco
Registry updated 17 Mar 2026
Registry record: ClinicalTrials.govNo phase23 sites
RecruitingSpain
NCT05992532
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Fundación Española de Hematología y Hemoterapía
Registry updated 11 Feb 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT03333200
Longitudinal Study of Neurodegenerative Disorders
University of Pittsburgh
Registry updated 9 Feb 2026
Registry record: ClinicalTrials.govPhase 1/23 sites
RecruitingChina
NCT06818838
A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
Lingyi Biotech Co., Ltd.
Pre-treatment window Registry sentence: “Newly treated or untreated GD1 patients who meet one or more of the following criteria at screening:”
Registry updated 26 Jan 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingArgentina
NCT06573723
Institutional Registry of Rare Diseases
Hospital Italiano de Buenos Aires
Registry updated 14 Jan 2026
Registry record: ClinicalTrials.govNo phase1 site
RecruitingUnited States
NCT04528355
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
Paul Szabolcs
Registry updated 13 Jan 2026
Registry record: ClinicalTrials.govPhase 21 site
RecruitingUnited States
NCT01962415
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
Paul Szabolcs
Registry updated 15 Dec 2025
Registry record: ClinicalTrials.gov
The same sites as a list
- United States17 open trials
- China5 open trials
- United Kingdom4 open trials
- Argentina3 open trials
- Israel3 open trials
- Spain3 open trials
- Netherlands2 open trials
- Brazil2 open trials
- Australia2 open trials
- Canada2 open trials
- Saudi Arabia2 open trials
- India2 open trials
- Pakistan2 open trials
- Taiwan2 open trials
- Lebanon1 open trials
- France1 open trials
- Serbia1 open trials
- Romania1 open trials
- South Korea1 open trials
- South Africa1 open trials
- Germany1 open trials
- Switzerland1 open trials
- Indonesia1 open trials
- Croatia1 open trials
- Philippines1 open trials
- Hungary1 open trials
- Vietnam1 open trials
- Kuwait1 open trials
- Peru1 open trials
- Bulgaria1 open trials
- Belgium1 open trials
- Italy1 open trials
- Japan1 open trials
- Thailand1 open trials
Who treats this
Institutions with trials or publications in this disease (OpenAlex, registries). Institutions only, no people.
All India Institute of Medical Sciences Bhubaneswar
Bhubaneswar, India
0 trials · 4 publications · ROR
Amsterdam Gastroenterology Endocrinology Metabolism
Amsterdam, Netherlands
0 trials · 4 publications · ROR
Biomedical Research Networking Center on Neurodegenerative Diseases
Madrid, Spain
0 trials · 3 publications · ROR
Cambridge University Hospitals NHS Foundation Trust
Cambridge, United Kingdom
0 trials · 3 publications · ROR
Centre Hospitalier Universitaire de Clermont-Ferrand
Clermont-Ferrand, France
0 trials · 9 publications · ROR
Chinese Academy of Medical Sciences & Peking Union Medical College
Beijing, China
0 trials · 4 publications · ROR
Cincinnati Children's Hospital Medical Center
Cincinnati, United States
1 trials · 12 publications · ROR
Clonal Heterogeneity, Leukemic environment, ThErapy Resistance of chronic leukemias
Clermont-Ferrand, France
0 trials · 7 publications · ROR
Consejo Nacional de Investigaciones Científicas y Técnicas
Buenos Aires, Argentina
0 trials · 5 publications · ROR
Federal Almazov North-West Medical Research Centre
St Petersburg, Russia
0 trials · 5 publications · ROR
First Pavlov State Medical University of St. Petersburg
St Petersburg, Russia
0 trials · 4 publications · ROR
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, Italy
1 trials · 6 publications · ROR
Fundacion Agencia Aragonesa para la Investigacion y el Desarrollo
Zaragoza, Spain
0 trials · 5 publications · ROR
Fundación para la Lucha contra las Enfermedades Neurológicas de la Infancia
Buenos Aires, Argentina
0 trials · 4 publications · ROR
King Edward Memorial Hospital and Seth G.S. Medical College
Mumbai, India
0 trials · 10 publications · ROR
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, United States
0 trials · 22 publications · ROR
National Institute of Neurological Disorders and Stroke
Bethesda, United States
0 trials · 7 publications · ROR
Post Graduate Institute of Medical Education and Research
Chandigarh, India
0 trials · 11 publications · ROR
University of Arkansas for Medical Sciences
Little Rock, United States
0 trials · 8 publications · ROR
University of North Carolina at Chapel Hill
Chapel Hill, United States
0 trials · 3 publications · ROR
What changed in the last 7 days
Registry facts, one per line, newest first.
Opened for recruitment
A Gaucher Disease Gene Therapy Trial With FLT201
NCT07223944 · Phase 3 · Argentina, Australia, Brazil, Canada, Spain, United Kingdom
Source: ClinicalTrials.govStatus changed: Completed → Running, not recruiting
NCT02843035 · Phase 2 · Germany, United Kingdom, Japan, United States
Source: ClinicalTrials.govStatus changed: Recruiting → Not yet recruiting
A Gaucher Disease Gene Therapy Trial With FLT201
NCT07223944 · Phase 3 · Argentina, Australia, Brazil, Canada, Spain, United Kingdom
Source: ClinicalTrials.gov
Results card
Trials that finished or stopped in the last 3 years, with the reason the registry gives and linked publications.
No phaseEnded 30 Jun 2026
No phaseEnded 30 Mar 2026
Phase 2Ended 31 Dec 2025
Completed33 participants
NCT02583672
Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
Phase 3Ended 26 Dec 2025
Completed57 participants
NCT03485677
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
No phaseEnded 2 Jun 2025
Phase 1Ended 4 Dec 2024
No phaseEnded 25 Nov 2024
Completed60 participants
NCT04845958
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
No phaseEnded 31 Oct 2024
Completed122 participants
NCT05641103
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
No phaseEnded 26 Aug 2024
Phase 3Ended 5 Aug 2024
Completed20 participants
NCT05529992
A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
No phaseEnded 24 May 2024
Completed125 participants
NCT05702814
A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition
No phaseEnded 14 May 2024
No phaseEnded 30 Mar 2024
Completed80 participants
NCT04997772
Digital Health Platform Customized for Patients With Gaucher Disease
Not medical advice. fetched 6 Nov 2026