Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
NCT00004293 · Status: UNKNOWN · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 24
Last updated 2005-06-24
Summary
OBJECTIVES:
I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.
Conditions
- Gaucher's Disease
Interventions
- DRUG
-
glucocerebrosidase
Sponsors & Collaborators
-
University of Pittsburgh
collaborator OTHER -
National Center for Research Resources (NCRR)
lead NIH
Principal Investigators
-
John Barranger · University of Pittsburgh
Study Design
- Purpose
- TREATMENT
Eligibility
- Min Age
- 18 Years
- Max Age
- 65 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 1999-11-30
Countries
- United States
Study Locations
More Related Trials
-
A Long-term Follow-up Study of Gaucher Disease
NCT03190837 ·Status: RECRUITING
-
Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
NCT00351156 ·Status: COMPLETED
-
Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
NCT01136304 ·Status: COMPLETED
-
A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease
NCT00465062 ·Status: COMPLETED
-
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
NCT00358150 ·Status: COMPLETED ·Phase: PHASE2
-
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
NCT00635427 ·Status: COMPLETED ·Phase: PHASE3
-
Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
NCT00962260 ·Status: NO_LONGER_AVAILABLE
-
A Screening Study Evaluating Disease Status of Gaucher Type I Patients
NCT00795197 ·Status: WITHDRAWN
-
A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease
NCT00430625 ·Status: COMPLETED ·Phase: PHASE3
-
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
NCT00705939 ·Status: COMPLETED ·Phase: PHASE3
-
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
NCT02843035 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2
-
Evaluate the Safety and Exploratory Efficacy of GC1119
NCT01653444 ·Status: COMPLETED ·Phase: PHASE1
-
Identification of Undiagnosed Gaucher Disease
NCT01716741 ·Status: UNKNOWN ·Phase: NA
-
A Gene Therapy Study in Patients With Gaucher Disease Type 1
NCT05324943 ·Status: COMPLETED ·Phase: PHASE1
-
A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
NCT01422187 ·Status: COMPLETED ·Phase: PHASE3
-
Gaucherite - A Study to Stratify Gaucher Disease
NCT03240653 ·Status: RECRUITING
-
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
NCT05422482 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1
-
Oral Supplementation of Gangliosides to Treat a Rare Metabolic Disorder
NCT02234024 ·Status: UNKNOWN ·Phase: NA
-
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
NCT00891202 ·Status: COMPLETED ·Phase: PHASE3
-
Long Term Follow-Up for Safety of AVR-RD-02
NCT06488261 ·Status: ACTIVE_NOT_RECRUITING
-
Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)
NCT00258778 ·Status: COMPLETED ·Phase: PHASE1
-
Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease
NCT00319046 ·Status: COMPLETED ·Phase: PHASE3
-
Longitudinal Studies of the Glycoproteinoses
NCT01891422 ·Status: COMPLETED
-
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
NCT04836377 ·Status: TERMINATED
-
Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-naïve Adult Male Patients With Fabry Disease
NCT02228460 ·Status: COMPLETED ·Phase: PHASE2