Phase 2 N=16 A Study of Fabrazyme in Pediatric Patients With Fabry Disease
Fabry Disease
Primary: Globotriaosylceramide (GL-3) Clearance in Capillary Endothelium in the Skin — 2; 14; 5; 0 patients
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Phase 4 N=82 A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease
Fabry Disease
Primary: Number of Participants Experiencing a Clinically Significant Renal, Cardiac or Cerebrovascular Event and/or Death in Fabrazyme (Agalsidase Beta) Patients as Compared to…
Phase 2 N=21 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Fabry Disease
Primary: Globotriaosylceramide (GL-3) Clearance in Kidney Interstitial Capillary Endothelium — 3; 21; 18; 17 Participants
Phase 4 N=67 A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Fabry Disease
Primary: Difference in Inverse Serum Creatinine Within Patients' Slopes Between the Placebo AGAL-008-00 (NCT00074984) and Fabrazyme AGAL02503 (NCT00081497) Periods — -0.044…
Phase 2 N=18 Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
Fabry Disease
Primary: Adverse Events — 54; 11; 29; 14 adverse events
Phase 3 N=67 Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease
Fabry Disease
Primary: Percentage Of Participants With At Least A 50% Reduction From Baseline To Month 6 In The Average Number Of Kidney Interstitial Capillary (IC) Globotriaosylceramide…
Phase 3 N=60 Study to Compare the Efficacy and Safety of Oral AT1001 and Enzyme Replacement Therapy in Patients With Fabry Disease
Fabry Disease
Primary: Annualized Rate Of Change From Baseline To Month 18 In Measured Glomerular Filtration Rate — -4.35; -3.24 mL/min/1.73 m^2/year
Phase 2 N=11 Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
Fabry Disease
Primary: Change From Baseline at Week 26 in Skin Globotriaosylceramide (GL-3) Score in Superficial Skin Capillary Endothelium: Number of Participants in Categories of Shift in…
Phase 2 N=17 Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Fabry Disease
Primary: Patients Who Experienced At Least One Adverse Event (AE) — 17 participants
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 2 N=12 Dosing Study of Replagal in Patients With Fabry Disease
Fabry Disease
Primary: Estimated Glomerular Filtration Rate (eGFR) — -0.66; -0.32 ml/min/month — p=0.01
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 2 N=9 A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Fabry Disease
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 2 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 2 N=4 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Fabry Disease
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 1 Participants
Phase 3 N=16 Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease
Fabry Disease
Primary: Incidence of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and AEs (Adverse Events) Leading to Discontinuation of Study Drug — 13; 1; 0…
Phase 2 N=9 A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Female Participants With Fabry Disease
Fabry Disease
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 0; 0; 0 Participants
Phase 2 N=5 A 24-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Fabry Disease
Primary: Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs) — 0 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 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 3 N=171 A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease
Fabry Disease
Primary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 163; 74; 7 Participants
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)
N/A N=82 A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.
Fabry Disease
Primary: Total Time Spent by Healthcare Professionals (HCPs) in the Preparation/Administration of a Single Dose of ERT in Patients With Fabry Disease — 151.9 minutes
Phase 3 N=118 Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
Fabry Disease
Primary: Neuropathic Pain Monthly Score: Change From Baseline to Month 6 — -1.64; -2.05 score on a scale — p=0.3189
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=6 A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Fabry Disease
Primary: Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156 — -3.31; -6.34 percent change
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 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 2 N=33 Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
Fabry Disease
Primary: Incidence of Treatment-emergent Adverse Events (TEAEs) - All — 2; 2; 3; 2 Participants