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NCT00004293 Clinical Trial

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Gaucher's Disease Clinical Trial

Clinical Trial Details — Status: Recruiting

Administrative data
NCT number NCT00004293
Other study ID # 199/11725
Secondary ID UPITTS-M1230
Status Recruiting
Phase Phase 2
First received October 18, 1999
Last updated June 23, 2005
Start date November 1999

Study information
Verified date December 2003
Source National Center for Research Resources (NCRR)
Contact n/a
Is FDA regulated No
Health authority United States: Federal Government
Study type Interventional

Clinical Trial Summary

OBJECTIVES:

I. Evaluate the efficacy and toxicity of glucocerebrosidase enzyme therapy in patients with Gaucher disease.

Description:

PROTOCOL OUTLINE:

Patients are treated with intravenous glucocerebrosidase every 2 weeks. The dose is based on clinical severity of disease and response to therapy.

Recruitment information / eligibility
Status Recruiting
Enrollment 24
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 65 Years
Eligibility PROTOCOL ENTRY CRITERIA:

--Disease Characteristics-- Gaucher disease with glucocerebrosidase deficiency confirmed by enzymatic or molecular assay At least 3 organ systems affected, based on the following criteria: Anemia Thrombocytopenia Organomegaly Bone deterioration on radiograph Pulmonary compromise Symptoms compromise daily activities or risk longevity No neurologic disease

Study Design

Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Drug:
• glucocerebrosidase

Locations
Country Name City State
United States University of Pittsburgh Pittsburgh Pennsylvania

Sponsors (2)
Lead Sponsor Collaborator
National Center for Research Resources (NCRR) University of Pittsburgh

Country where clinical trial is conducted

United States, 

See also
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Completed NCT00004488 - Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease Phase 2
Completed NCT00001410 - PEG-Glucocerebrosidase for the Treatment of Gaucher Disease Phase 1
Completed NCT00004294 - Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease Phase 1
Completed NCT04656600 - Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ Phase 4
Completed NCT01427517 - Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease Phase 1
Completed NCT00176904 - Stem Cell Transplant for Inborn Errors of Metabolism Phase 2/Phase 3
Completed NCT00001234 - Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells Phase 1
Completed NCT06188325 - A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers Phase 1
Completed NCT00001416 - Bone Response to Enzyme Replacement in Gaucher's Disease Phase 2
Completed NCT06193304 - A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth Phase 1
Active, not recruiting NCT00005900 - Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT01586455 - Human Placental-Derived Stem Cell Transplantation Phase 1