GlucocerebrosidasePhase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Sponsor:

National Center for Research Resources (NCRR)

Code:

NCT00004293

Conditions

Gaucher's Disease

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

Interventions

glucocerebrosidase

Study Details

Brief summary:

OBJECTIVES:

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

Conditions

Gaucher's Disease

Study ID

NCT00004293

Start date

Nov, 1999

Status verified date

Dec, 2003

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Not accepted

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

Enrollment

24 participants

Primary purpose

Treatment

Interventions and Outcome Measures

Interventions

glucocerebrosidase

Central Contacts and Locations

Locations

University of Pittsburgh

Recruiting

Pittsburgh, Pennsylvania, United States, 15260

Contacts

John Barranger

412-624-4623

More Information

Sponsor

National Center for Research Resources (NCRR)

Last update posted

Jun 24, 2005

Last verified

Dec, 2003

Keywords

Gaucher's disease
inborn errors of metabolism
rare disease
sphingolipidoses

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by National Center for Research Resources (NCRR) on 2005-06-24.