Phase 1 N=6 A Gene Therapy Study in Patients With Gaucher Disease Type 1
Gaucher Disease, Type 1
Primary: Number of Participants With Treatment-emergent Adverse Events Over Time — 6; 6; 6; 2 Participants
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Phase 2 N=26 A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
Gaucher Disease, Type 1 · Cerebroside Lipidosis Syndrome · Glucocerebrosidase Deficiency Disease
Primary: Percentage of Participants Demonstrating A Meaningful Clinical Response — 77 percentage of participants
Phase 3 N=6 Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Gaucher Disease
Primary: Number of Severe Adverse Events (SAE) — 1 events
Phase 3 N=25 A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
Gaucher Disease, Type 1
Primary: Change From Baseline to 12 Months in Hemoglobin Concentration for the 60 U/kg Treatment Group. — 2.429 g/dL — p=<0.0001
Phase 3 N=34 Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
Gaucher Disease, Type 1
Primary: Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. — 1.624; 1.488 gram per deciliter (g/dl)
Phase 2 N=7 Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
Gaucher Disease, Type 3
Primary: Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration — 2.15 g/dL
Phase 3 N=40 Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Gaucher Disease
Primary: Participants Who Experienced at Least One Adverse Event — 34 participants
Phase 2 N=10 Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
Gaucher Disease
Primary: Evaluation of Long Term Safety — 0; 10; 1; 1 Participants
Phase 3 N=5 Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease
Gaucher Disease
Primary: Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs) — 2; 0; 2 participants
Phase 3 N=8 Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
Gaucher Disease, Type 1
Primary: The Difference in Hemoglobin Concentration [g/dL] — 9.49; 11.44 g/dL
N/A N=60 Auto-antibodies Prevalence and CD1 Role in Gaucher Disease
Gaucher Disease
Primary: Number of Patients With GD Diagnosis Confirmed by : Enzyme Testing of acidβ-glucosidase Activity Activity <15% in Blood Leucocytes Completed When Necsssary by GB1…
Phase 3 N=44 Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher Disease
Primary: Spleen Volume — 2324.0; 2120.0; 778.0; 1707.7 mL
Phase 3 N=95 An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
Gaucher Disease, Type 1
Primary: Overall Summary of Treatment Emergent Adverse Events — 3; 1; 3; 38 Participants
Phase 3 N=32 A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher Disease
Primary: Change From Baseline in Spleen Volume Measured by MRI. — -26.91; -38.01 percentage of change — p=<0.0001
Phase 4 N=12 Growth and Development Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Recumbent Height/Length of Participants in Centimeters (cm) — 80.4; 93.8; 67.7; 91.1 cm
Phase 3 N=20 A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease
Gaucher Disease
Primary: Percentage of Participants With at Least One Serious Treatment-Emergent Adverse Event (TEAE) — 20 percentage of participants
Phase 4 N=14 A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Gaucher Disease, Type 3
Primary: Percent Change From Baseline in Spleen Volume Measured by MRI — -51.33 Percent change
Phase 4 N=4 A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy
Gaucher Disease
Primary: Number of Participants With Clinically Stable Hemoglobin (Hb) Concentration From Baseline up to Month 12 — 2; 2 Participants
Phase 3 N=42 Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
Gaucher Disease Type 1
Primary: Liver Volume at Baseline and at End of Treatment — 1774.6; 1727.1 cm^3
Phase 3 N=40 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
Gaucher Disease, Type 1
Primary: PAP: Percent Change From Baseline in Spleen Volume (in Multiples of Normal [MN]) at Week 39 of the Primary Analysis Period With Eliglustat Tartrate Treatment as Compared…
Phase 2 N=19 A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
Gaucher Disease · Type 1 Gaucher Disease · Gaucher Disease, Type 1
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 2; 0 Participants
Phase 2 N=30 A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
Gaucher Disease, Type 1 · Type 1 Gaucher Disease · Gaucher Disease
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 0; 0; 0; 0 Participants
Phase 4 N=21 Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease
Gaucher Disease
Primary: Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-Score up to End of Study (EOS) (Week 103) — 0.17 Z-score
Phase 2 N=13 Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease
Pompe Disease
Primary: Safety Assessments of the rAAV1-CMV-GAA (Study Agent), Changes Post Study Agent Administration. — 40,031; 29,638; 5,509,882; 1,907,161 mU/mL
Phase 4 N=13 High Dose or High Dose Frequency Study of Alglucosidase Alfa
Pompe Disease · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis 2 Acid Maltase Deficiency
Primary: Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment — 0; 0; 0; 1…
Phase 4 N=11 A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
Gaucher Disease
Primary: Hemoglobin — 1.2; 0.4; 1.1; 1.5 g/dL
Phase 1 N=9 Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
Parkinson's Disease · Gaucher's Disease
Primary: Brain GSH — 55; 41; 34 percent increase from baseline
Phase 3 N=90 A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
Pompe Disease (Late-onset) · Glycogen Storage Disease Type II (GSD-II) · Acid Maltase Deficiency Disease
Primary: Summary of Patients Reporting Treatment-Emergent Adverse Events — 60; 30; 32; 17 participants
Phase 4 N=95 Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
Gaucher Disease, Type 1 · Cerebroside Lipidosis Syndrome · Glucocerebrosidase Deficiency Disease
Primary: Number of Participants With Clinical Success at Month 24/Discontinuation — 21; 36; 0.808; 0.632 patients
Phase 4 N=90 Late-Onset Treatment Study Extension Protocol
Pompe Disease (Late-Onset) · Glycogen Storage Disease Type II (GSD-II) · Glycogenesis Type II
Primary: Summary of Participants Reporting Treatment-Emergent Adverse Events For Participants Treated With Alglucosidase Alfa During Study AGLU02704 (NCT00158600) — 60; 37; 21…