Gaucher Disease
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-04-06Clinical Trial Update: Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
NHGRI-sponsored clinical trial evaluating positron emission tomography imaging in people with Gaucher mutations (including those with Parkin…
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CLINICAL_TRIALS trial_result · 2026-04-03Clinical Trial Update: High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3) [NA]
The trial evaluating high-dose ambroxol for pediatric Gaucher disease type 3 has been terminated.
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
Clinical trial NCT05641103 (PREDIGA 2), an Educational and Diagnostic Project for Gaucher disease and acid sphingomyelinase deficiency, has …
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ [Phase 4]
A Phase 4 study assessing the efficacy and safety of imiglucerase in Chinese patients with Gaucher disease type III has been completed.
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CLINICAL_TRIALS trial_result · 2025-07-22Clinical Trial Update: Long Term Follow-Up for Safety of AVR-RD-02
This is a long-term follow-up safety study of AVR-RD-02 for Gaucher disease, currently listed as active but not recruiting.
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CLINICAL_TRIALS trial_result · 2025-05-31Clinical Trial Update: The GBA Multimodal Study in Parkinson's Disease
The GBA Multimodal Study in Parkinson's Disease is an ongoing clinical trial investigating GBA gene mutations in Parkinson's disease and is …
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CLINICAL_TRIALS trial_result · 2025-02-26Clinical Trial Update: A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease [Phase 3]
Phase 3 trial update of Velaglucerase alfa (VPRIV) in Chinese children, adolescents, and adults with Type 1 Gaucher disease has completed, w…
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CLINICAL_TRIALS trial_result · 2025-01-10Clinical Trial Update: Survey Study for Velaglucerase Alfa (VPRIV) in Japan
A survey study evaluating velaglucerase alfa (VPRIV) in Gaucher disease patients in Japan has completed, and the results have been posted.
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CLINICAL_TRIALS trial_result · 2024-12-17Clinical Trial Update: A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease [Phase 4]
A Phase 4 multicenter trial evaluating the safety and efficacy of taliglucerase alfa in subjects with Gaucher disease, Type 3, has completed…
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CLINICAL_TRIALS trial_result · 2024-12-11Clinical Trial Update: A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition
Takeda-sponsored study assessing substances potentially indicating bone problems in adults with Gaucher disease has completed.
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CLINICAL_TRIALS trial_result · 2024-01-18Clinical Trial Update: Role of Vitamin E in Gaucher Disease Patients [Phase 3]
Phase 3 clinical trial evaluating role of Vitamin E in Gaucher disease patients has completed.
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CLINICAL_TRIALS trial_result · 2024-01-18Clinical Trial Update: Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease [Phase 1, Phase 2]
A phase 1/2 trial of AVR-RD-02 using a lentiviral vector in Gaucher disease has been terminated with results posted.
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CLINICAL_TRIALS trial_result · 2023-12-21Clinical Trial Update: A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy [Phase 4]
Phase 4 trial updating enzyme replacement therapy VPRIV in people with Gaucher disease previously treated with substrate reduction therapy; …
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CLINICAL_TRIALS trial_result · 2022-07-15Clinical Trial Update: Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies [Phase 3]
Phase 3b trial update evaluating skeletal response to eliglustat in adults who completed prior studies for Gaucher disease; status completed…
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CLINICAL_TRIALS trial_result · 2022-02-01Clinical Trial Update: Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease [Phase 4]
A Phase 4 study evaluating velaglucerase alfa (VPRIV) on bone-related pathology in treatment-naïve adults with Type 1 Gaucher disease has co…
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CLINICAL_TRIALS trial_result · 2018-10-30Clinical Trial Update: Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease. [Phase 4]
A Phase 4 clinical trial evaluating chronic hepatitis C treatment in Egyptian children with Gaucher disease is ongoing, with status currentl…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy [Phase 2]
Phase 2 trial evaluating AT2101 (afegostat tartrate) in adults with Type 1 Gaucher disease already on enzyme replacement therapy has complet…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients [Phase 2]
Long-term extension study update of AT2101 (Afegostat Tartrate) in patients with Type 1 Gaucher disease completed, with results posted in 20…
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CLINICAL_TRIALS trial_result · 2018-08-15Clinical Trial Update: A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease [Phase 2]
Phase 2 trial update for oral AT2101 (afegostat tartrate) in treatment-naive Gaucher disease patients completed with posted results.
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease [Phase 2]
This update notes that the Phase II randomized, controlled trial of OGT 918 for Gaucher disease (neuronopathic form) has completed.
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CLINICAL_TRIALS trial_result · 2017-07-02Clinical Trial Update: Effects of Enzyme Replacement in Gaucher's Disease
Completed clinical trial evaluating enzyme replacement therapy effects in Gaucher disease. The provided snippet does not detail specific res…
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CLINICAL_TRIALS trial_result · 2017-04-04Clinical Trial Update: Exploration of Immunity in Gaucher Disease
This is a completed clinical trial exploring immunity in Gaucher disease sponsored by O & O Alpan LLC (NCT01358188).
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CLINICAL_TRIALS trial_result · 2017-03-08Clinical Trial Update: A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate [Phase 1]
This Phase 1 study evaluated the effects of renal impairment on the pharmacokinetics and tolerability of eliglustat tartrate in Gaucher dise…
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CLINICAL_TRIALS trial_result · 2017-02-10Clinical Trial Update: A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate [Phase 1]
Phase 1 study evaluating the effect of hepatic impairment on the pharmacokinetics and tolerability of eliglustat tartrate in Gaucher disease…
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CLINICAL_TRIALS trial_result · 2016-12-02Clinical Trial Update: A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE) [Phase 3]
Phase 3 trial comparing once-daily versus twice-daily dosing of eliglustat tartrate in Gaucher disease has completed with results posted.