Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Status

UNKNOWN

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00004293

Enrollment

Registered

1999-10-19

Start date

1999-11-30

Completion date

Unknown

Last updated

2005-06-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Gaucher's Disease

Keywords

Gaucher's disease, inborn errors of metabolism, rare disease, sphingolipidoses

Brief summary

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

Detailed 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.

Interventions

DRUGglucocerebrosidase

Study design

Primary purpose

TREATMENT

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion criteria

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026