Suspended

Intravenous Glucocerebrosidase for Gaucher Disease Treatment

0 criteria met from your profileSee at a glance how your profile meets each eligibility criteria.
What is being tested

glucocerebrosidase

Drug
Who is being recruted

Brain Diseases+18

+ Brain Diseases, Metabolic

+ Central Nervous System Diseases

From 18 to 65 Years
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 2
Interventional
Study Start: November 1999
See protocol details

Summary

Principal SponsorNational Center for Research Resources (NCRR)
Study ContactJohn Barranger
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Study start date: November 1, 1999

Actual date on which the first participant was enrolled.

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

NCT00004293
Principal SponsorNational Center for Research Resources (NCRR)
Study ContactJohn Barranger
Last updated: June 24, 2005
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

24 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 65 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Brain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesLipid Metabolism, Inborn ErrorsLipidosesMetabolic DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesPathologic ProcessesPathological Conditions, Signs and SymptomsLysosomal Storage DiseasesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornDisease AttributesGenetic Diseases, InbornLipid Metabolism DisordersGaucher DiseaseMetabolism, Inborn ErrorsSphingolipidosesRare Diseases

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

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Recruiting

University of Pittsburgh

Pittsburgh, United StatesOpen University of Pittsburgh in Google Maps
SuspendedOne Study Center