Gaucher Disease, Type 1 Clinical Trials

38 clinical trials for Gaucher Disease, Type 1 across international registries, including 6 currently recruiting participants. Compare status, phase, eligibility criteria and locations.

• A Gaucher Disease Gene Therapy Trial With FLT201 — RECRUITING · PHASE3
• A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) — RECRUITING · PHASE1
• Prodromal Parkinsonian Features in GBA1 Mutation Carriers — RECRUITING
• A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease — RECRUITING · PHASE1
• A PHASE 3 SAFETY AND EFFICACY TRIAL OF FLT201 GENE THERAPY IN PATIENTS WITH GAUCHER DISEASE TYPE 1 — RECRUITING · PHASE3
• AN ADAPTIVE, OPEN-LABEL, MULTINATIONAL PHASE 1/2 STUDY OF THE SAFETY AND EFFICACY OF EX VIVO, LENTIVIRAL VECTOR-MEDIATED GENE THERAPY AVR-RD-02 FOR SUBJECTS WITH TYPE 1 GAUCHER DISEASE — RECRUITING · PHASE1
• Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension — ACTIVE_NOT_RECRUITING · PHASE2
• A Gene Therapy Study in Patients With Gaucher Disease Type 1 — COMPLETED · PHASE1
• A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects with Gaucher Disease (GALILEO-2) — TERMINATED · PHASE2
• Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) — COMPLETED · PHASE2
• Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) — ACTIVE_NOT_RECRUITING · PHASE1
• Baby Detect : Genomic Newborn Screening — COMPLETED
• Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease — COMPLETED · PHASE3
• A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy. — COMPLETED · PHASE2
• Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy — COMPLETED · PHASE2
• Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 — TERMINATED · PHASE2
• Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) — COMPLETED · PHASE4
• Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs — COMPLETED · NA
• Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease — OTHER · PHASE1
• A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease — COMPLETED · PHASE3
• 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) — COMPLETED
• An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease — COMPLETED · PHASE3
• Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease — COMPLETED · PHASE3
• Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease — OTHER
• Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease — OTHER
• A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy — COMPLETED · PHASE2
• A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients — COMPLETED · PHASE2
• A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease — COMPLETED · PHASE2
• The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease — WITHDRAWN · PHASE4
• OPEN LABEL, TWO COHORT (WITH AND WITHOUT IMIGLUCERASE), MULTICENTER STUDY TO EVALUATE PHARMACOKINETICS (PK), SAFETY, AND EFFICACY OF ELIGLUSTAT IN PEDIATRIC PATIENTS WITH GAUCHER DISEASE (GD) TYPE 1 (GD1) AND TYPE 3 (GD3) — COMPLETED
• Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease — COMPLETED · PHASE3
• A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) — COMPLETED · PHASE3
• A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients — COMPLETED · PHASE2
• A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) — COMPLETED · PHASE3
• OPEN LABEL INTERVENTIONAL MULTICENTER PHASE 3B STUDY TO EVALUATE SKELETAL RESPONSE TO ELIGLUSTAT IN ADULT PATIENTS WHO SUCCESSFULLY COMPLETED THE PHASE 2 OR PHASE 3 STUDIES. — COMPLETED · PHASE3
• A Screening Study Evaluating Disease Status of Gaucher Type I Patients — WITHDRAWN
• Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease — COMPLETED · PHASE4
• Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease — COMPLETED · NA

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