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
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
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=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=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 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 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 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 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=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 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
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 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 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
Phase 2 N=6 A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 174; 355; 0; 1 events
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=6 A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
Sanfilippo Syndrome Type A (MPS IIIA)
Primary: Safety as Measured by Adverse Events Frequencies (by Type and Severity) — 53; 128; 0; 1 number of events
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 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=31 Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Gaucher Disease
Primary: Number of Participants With Mobility Status Assessments at Study Baseline, Weeks 52, 104, 156 and 208 — 31; 0; 0; 0 Participants
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 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=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 2 N=21 Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo Syndrome
Primary: Number of Participants With Overall Response Using Bayley Scales of Infant Development Assessment Third Edition (BSID-III) — 0; 2; 1 participants — p=0.4615
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 2 N=12 Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Mucopolysaccharidosis (MPS)
Primary: Number of Treatment Emergent Serious Adverse Events (SAE) — 5; 3; 2 events
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 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 3 N=160 A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
Gaucher Disease, Type 1
Primary: Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period — 84.8; 93.6 percentage of participants
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 2 N=11 Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB)
Mucopolysaccharidosis IIIB
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1; 0; 1 Participants