Gaucher Disease Clinical Trials

90 clinical trials for Gaucher Disease across international registries, including 14 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• Advancing Integrated Therapies for Gaucher Disease — RECRUITING
• Genetic Studies of Lysosomal Storage Disorders — RECRUITING
• ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program — RECRUITING
• A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) — RECRUITING · PHASE1
• Gaucher Disease Outcome Survey (GOS) — RECRUITING
• World Data on Ambroxol for Patients With GD and GBA Related PD — RECRUITING
• International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry — RECRUITING
• Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease — RECRUITING
• GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma — RECRUITING
• Longitudinal Study of Neurodegenerative Disorders — RECRUITING
• Institutional Registry of Rare Diseases — RECRUITING
• A Long-term Follow-up Study of Gaucher Disease — RECRUITING
• The GBA Multimodal Study in Parkinson's Disease — RECRUITING
• FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases — RECRUITING
• Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations — COMPLETED
• A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease — ACTIVE_NOT_RECRUITING · PHASE1
• A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion — NOT_YET_RECRUITING
• Survey Study for Velaglucerase Alfa (VPRIV) in Japan — COMPLETED
• PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD" — COMPLETED
• Long Term Follow-Up for Safety of AVR-RD-02 — ACTIVE_NOT_RECRUITING
• Omics Gaucher Study: Multiomic Approach — ACTIVE_NOT_RECRUITING
• A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease — COMPLETED · PHASE3
• Drug Discovery for Parkinson's With Mutations in the GBA Gene — OTHER
• A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition — COMPLETED
• 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) — COMPLETED · NA
• Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3 — OTHER · PHASE2
• Digital Health Platform Customized for Patients With Gaucher Disease — COMPLETED
• Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency — NOT_YET_RECRUITING
• A Study of Velaglucerase Alfa (VPRIV) Given as Standard Patient Care in Young Children With Gaucher Disease — COMPLETED
• Biomarkers Related to Bone in Pediatric Gaucher Disease — OTHER
• A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies — TERMINATED
• Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease — TERMINATED · PHASE1
• Role of Vitamin E in Gaucher Disease Patients — COMPLETED · PHASE3
• A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy — COMPLETED · PHASE4
• Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India — COMPLETED
• A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease — COMPLETED · PHASE3
• Assessment of Neurological Manifestations in Gaucher Disease Patients — OTHER
• Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography — TERMINATED
• Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies — COMPLETED · PHASE3
• A National Study in Patients With Unexplained Splenomegaly — COMPLETED
• Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease — COMPLETED · PHASE4
• Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease — COMPLETED · PHASE3
• Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) — COMPLETED · PHASE1
• Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease — COMPLETED · PHASE3
• Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase — COMPLETED · PHASE2
• Complement Activation in the Lysosomal Storage Disorders — WITHDRAWN
• Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease — COMPLETED
• Getting Global Rare Disease Insights Through Technology Study — OTHER · NA
• A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers — COMPLETED · PHASE1
• Genzyme Osteopenia/Osteoporosis Study — COMPLETED

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