Clinical Trials Logo
  1. Home
  2. Gaucher Disease
  3. NCT00391625

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Gaucher Disease Clinical Trial

Official title:
An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy

Clinical Trial Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the long term safety of enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.

Clinical Trial Description

Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the central nervous system (CNS). Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (the long term safety of enzyme replacement therapy with DRX008A (GA-GCB; velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB (velaglucerase alfa) contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT00391625
Study type Interventional
Source Takeda
Contact
Status Completed
Phase Phase 1/Phase 2
Start date September 13, 2004
Completion date January 31, 2008
View Details
See also
  Status Clinical Trial Phase
Withdrawn NCT04189601 - Complement Activation in the Lysosomal Storage Disorders
Completed NCT02536911 - A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate Phase 1
Completed NCT02536937 - A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate Phase 1
Completed NCT04430881 - A National Study in Patients With Unexplained Splenomegaly
Completed NCT01411228 - A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease Phase 3
Terminated NCT04094181 - A Study of VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies
Completed NCT03625882 - Survey Study for Velaglucerase Alfa (VPRIV) in Japan
Active, not recruiting NCT05526664 - Omics Gaucher Study: Multiomic Approach
Completed NCT02536755 - Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies Phase 3
Recruiting NCT01344096 - Thrombocytopathy in Gaucher Disease Patients N/A
Completed NCT01881633 - A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers Phase 1
Recruiting NCT06116071 - Biomarkers Related to Bone in Pediatric Gaucher Disease
Recruiting NCT01951989 - Intra-monocyte Imiglucerase Kinetics in Gaucher Disease Phase 2
Completed NCT00258778 - Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD) Phase 1
Recruiting NCT04388969 - World Data on Ambroxol for Patients With GD and GBA Related PD
Recruiting NCT05992532 - GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
Terminated NCT04145037 - Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease Phase 1/Phase 2
Completed NCT00302146 - Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations
Active, not recruiting NCT02605603 - SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease
Completed NCT02053896 - A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease Phase 2