NCT00004293

Brief Summary

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

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at below P25 for phase_2

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 18, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 19, 1999

Completed
13 days until next milestone

Study Start

First participant enrolled

November 1, 1999

Completed
Last Updated

June 24, 2005

Status Verified

December 1, 2003

First QC Date

October 18, 1999

Last Update Submit

June 23, 2005

Conditions

Gaucher's Disease

Keywords

Gaucher's diseaseinborn errors of metabolismrare diseasesphingolipidoses

Interventions

glucocerebrosidaseDRUG

Eligibility Criteria

Age18 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

Study Sites (1)

University of Pittsburgh

Pittsburgh, Pennsylvania, 15260, United States

RECRUITING

MeSH Terms

Conditions

Gaucher DiseaseMetabolism, Inborn ErrorsRare DiseasesSphingolipidoses

Interventions

Glucosylceramidase

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism DisordersDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

GlucosidasesGlycoside HydrolasesHydrolasesEnzymesEnzymes and Coenzymes

Study Officials

  • John Barranger

    University of Pittsburgh

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Purpose
TREATMENT
Sponsor Type
NIH

Study Record Dates

First Submitted

October 18, 1999

First Posted

October 19, 1999

Study Start

November 1, 1999

Last Updated

June 24, 2005

Record last verified: 2003-12

Locations

US(1)