Gaucher Disease: all 174 studies
Page 1 of 2. Open to participants first (40), then studies with results posted, then the rest; newest registration first within each.
- Parkinson's Disease in Type 1 Gaucher DiseaseRecruitingNCT07886879
- Long-Term Safety and Tolerability of Nizubaglustat in Participants With Rare Lysosomal DisordersNot yet recruitingNCT07885917Phase 2/3
- Study of Velaglucerase Beta ERT in Children With Gaucher DiseaseNot yet recruitingNCT07847411Phase 3
- Nutritional Status of Gaucher Disease PatientsNot yet recruitingNCT07845188
- Advancing Integrated Therapies for Gaucher DiseaseRecruitingNCT07758816
- Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)Not yet recruitingNCT07715084Phase 2
- Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).AvailableNCT07675031
- A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's DiseaseNot yet recruitingNCT07603050Phase 1/2
- A Gaucher Disease Gene Therapy Trial With FLT201RecruitingNCT07223944Phase 3
- A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher DiseaseRecruitingNCT06818838Phase 1/2
- Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3Enrolling by invitationNCT06627543
- Institutional Registry of Rare DiseasesRecruitingNCT06573723
- FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare DiseasesRecruitingNCT06539169
- Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3Not yet recruitingNCT06523517Phase 2
- An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher DiseaseRecruitingNCT06272149Early Phase 1
- Screening for Gaucher Disease and Acid Sphingomyelinase DeficiencyNot yet recruitingNCT06258577
- A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type IRecruitingNCT06162338Not applicable
- Biomarkers Related to Bone in Pediatric Gaucher DiseaseRecruitingNCT06116071
- GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple MyelomaRecruitingNCT05992532
- Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher DiseaseRecruitingNCT05843552
- Assessment of Neurological Manifestations in Gaucher Disease PatientsNot yet recruitingNCT05816993
- A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home InfusionNot yet recruitingNCT05669729
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
- MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)RecruitingNCT05586243
- Drug Discovery for Parkinson's With Mutations in the GBA GeneRecruitingNCT05536388
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT05368038
- Prodromal Parkinsonian Features in GBA1 Mutation CarriersRecruitingNCT05253560
- 4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating NeuroblastomaNot yet recruitingNCT04637503Phase 1/2
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)RecruitingNCT04532047Phase 1
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT04528355
- World Data on Ambroxol for Patients With GD and GBA Related PDRecruitingNCT04388969
- Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory OsteosarcomaRecruitingNCT03721068Phase 1
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT03333200
- Gaucher Disease Outcome Survey (GOS)RecruitingNCT03291223
- Gaucherite - A Study to Stratify Gaucher DiseaseRecruitingNCT03240653
- A Long-term Follow-up Study of Gaucher DiseaseRecruitingNCT03190837
- Oxidative Stress and Inflammatory Biomarkers in Gaucher DiseaseRecruitingNCT02437396
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 2
- International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registryRecruitingNCT00358943
- Genetic Studies of Lysosomal Storage DisordersEnrolling by invitationNCT00001215
- A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher DiseaseCompletedNCT05529992Phase 3Results posted
- A Gene Therapy Study in Patients With Gaucher Disease Type 1CompletedNCT05324943Phase 1Results posted
- A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction TherapyCompletedNCT04718779Phase 4Results posted
- Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher DiseaseTerminatedNCT04145037Phase 1/2Results posted
- A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher DiseaseCompletedNCT04002830Phase 4Results posted
- Survey Study for Velaglucerase Alfa (VPRIV) in JapanCompletedNCT03625882Results posted
- Phase III Study of ISU302 in Patients With Type 1 Gaucher DiseaseCompletedNCT02770625Phase 3Results posted
- Auto-antibodies Prevalence and CD1 Role in Gaucher DiseaseCompletedNCT02650219Results posted
- Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher DiseaseCompletedNCT02574286Phase 4Results posted
- Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 StudiesCompletedNCT02536755Phase 3Results posted
- Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher DiseaseCompletedNCT01842841Phase 3Results posted
- Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher DiseaseCompletedNCT01685216Phase 1/2Results posted
- Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher DiseaseCompletedNCT01614574Phase 3Results posted
- Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's DiseaseCompletedNCT01427517Phase 1Results posted
- A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher DiseaseCompletedNCT01422187Phase 3Results posted
- A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher DiseaseCompletedNCT01411228Phase 3Results posted
- A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher DiseaseCompletedNCT01132690Phase 4Results posted
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)CompletedNCT01074944Phase 3Results posted
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)CompletedNCT00943111Phase 3Results posted
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)CompletedNCT00891202Phase 3Results posted
- A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher PatientsCompletedNCT00813865Phase 2Results posted
- Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human GlucocerebrosidaseCompletedNCT00712348Phase 3Results posted
- Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension TrialCompletedNCT00705939Phase 3Results posted
- An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher DiseaseCompletedNCT00635427Phase 3Results posted
- Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher DiseaseCompletedNCT00553631Phase 3Results posted
- Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With ImigluceraseCompletedNCT00478647Phase 2/3Results posted
- A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher DiseaseCompletedNCT00446550Phase 2Results posted
- A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement TherapyCompletedNCT00433147Phase 2Results posted
- A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher DiseaseCompletedNCT00430625Phase 3Results posted
- Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)CompletedNCT00391625Phase 1/2Results posted
- A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher DiseaseCompletedNCT00376168Phase 3Results posted
- Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher DiseaseCompletedNCT00364858Phase 4Results posted
- A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher PatientsCompletedNCT00358150Phase 2Results posted
- Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher DiseaseCompletedNCT00319046Phase 3Results posted
- Stem Cell Transplant for Inborn Errors of MetabolismCompletedNCT00176904Phase 2/3Results posted
- High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)CompletedNCT07285369Not applicable
- Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)Active, not recruitingNCT06545136Phase 1/2
- A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher DiseaseActive, not recruitingNCT06528080Early Phase 1
- A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male ParticipantsActive, not recruitingNCT06517914Phase 1
- Long Term Follow-Up for Safety of AVR-RD-02Active, not recruitingNCT06488261
- Role of Vitamin E in Gaucher Disease PatientsCompletedNCT06211478Phase 3
- A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the MouthCompletedNCT06193304Phase 1
- A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor MetabolizersCompletedNCT06188325Phase 1
- A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male ParticipantsCompletedNCT06143904Phase 1
- A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.CompletedNCT06050967Phase 2
- Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)CompletedNCT05908656Not applicable
- An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3WithdrawnNCT05815004Phase 2/3
- A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher ConditionCompletedNCT05702814
- Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
- PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"CompletedNCT05641103
- Omics Gaucher Study: Multiomic ApproachActive, not recruitingNCT05526664
- A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)Active, not recruitingNCT05487599Phase 1/2
- Phase 1/2 Study of CAN103 in Subjects With Gaucher DiseaseStatus unknownNCT05447494Phase 1/2
- Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3Active, not recruitingNCT05222906Phase 3
- Digital Health Platform Customized for Patients With Gaucher DiseaseCompletedNCT04997772
- A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged SpleenCompletedNCT04845958
- A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02TerminatedNCT04836377
- A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy VolunteersCompletedNCT04787887Phase 1
- Getting Global Rare Disease Insights Through Technology StudyStatus unknownNCT04758130Not applicable
- A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher DiseaseCompletedNCT04721366