Trials and Research
Below is a list of Research and Trials which are being carried out into Gaucher Disease.
Trials and Research
01
Current Trials and Research
These trials are active and recruiting.
- A Long-term Follow-up Study of Gaucher Disease
- Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease
- Gaucher Disease Outcome Survey (GOS)
- Phase 1/2 Lentiviral Vector Gene Therapy – The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
- Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
- Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
- Gaucherite – A Study to Stratify Gaucher Disease
- Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
- GZ/SAR402671 in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3
- Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
- Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)
- International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
- Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease
- Liver and the Spleen Stifness in Adult Patients With Gaucher’s Disease Using Ultrasound Shear Wave Elastography
- Survey Study for Velaglucerase Alfa (VPRIV) in Japan
- Genzyme Osteopenia/Osteoporosis Study
- Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease
- Biomarker Analysis for GBA Associated Parkinson’s Disease
- Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
- The GBA Multimodal Study in Parkinson’s Disease
- Longitudinal Study of Neurodegenerative Disorders
- Molecular and Cellular Mechanisms of Lysosomal Storage Diseases
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
02
Planned Trials and Research
These trials are planned and not yet recruiting or active without recruitment.
- Miglustat on Gaucher Disease Type IIIB
- Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
- Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
- SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
- Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
- Eliglustat on Gaucher Disease Type IIIB
- A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
- Study of Skeletal Response to Eliglustat in Patients With Gaucher Disease
- Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
- Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
- Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India
- Canadian VPRIV® Study in Participants Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
- World Data on Ambroxol for Patients With GD and GBA Related PD
- Biomarker for Gaucher Disease: BioGaucher (BioGaucher)
- Therapeutic Exercise to Treat Neuropathic Pain
- A National Study in Patients With Unexplained Splenomegaly
- In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
- Complement Activation in the Lysosomal Storage Disorders
- Human Placental-Derived Stem Cell Transplantation
- Screening of Lysosomal Storage Disorders Diseases in Minority Groups
03
Completed Trials and Research
These trials have been completed and may have data.
- Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease
- A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
- A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
- Exploration of Immunity in Gaucher Disease
- Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
- Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
- Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
- A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease
- Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
- Home Therapy With VPRIV in Gaucher’s Disease
- Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
- A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)
- Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
- Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
- A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease
- Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease
- Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
- Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
- Bone Response to Enzyme Replacement in Gaucher’s Disease
- Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
- A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
- An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
- Macrophagic Activation and Immunophenotypic Profile of Patients With Gaucher’s Disease
- Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease
- Effects of Enzyme Replacement in Gaucher’s Disease
- OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
- Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
- PEG-Glucocerebrosidase for the Treatment of Gaucher Disease
- A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
- A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
- Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients
- Intravenous N-acetylcysteine for the Treatment of Gaucher’s Disease and Parkinson’s Disease
- Oxidative Stress-Related Biomarkers in Gaucher Disease: A Preliminary Study
- Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
- A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
- An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112
- Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
- A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
- Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
- A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers
- Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
- A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
- A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
- A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate
- Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher’s Disease
- Taste Evaluation of Different Liquid Formulations With Eliglustat
- Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
- Gene Therapy for Gaucher’s and Fabry Disease Using Viruses and Blood-Forming Cells
- A Multicenter Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.
- Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
- A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
- Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism
- MR Spectroscopy and Disease Severity Score for Gaucher in Pediatric Population
- Clinical and Pathophysiological Investigations Into Erdheim Chester Disease
- Stem Cell Transplant for Inborn Errors of Metabolism
- Study to Determine Mutations in the Gaucher Gene in Patients With Idiopathic Parkinson’s Disease for Phenotype-genotype Correlation