Completed

Clinical and Biochemical Effects of Macrophage-Targeted Glucocerebrosidase on Neurological Involvement in Neuronopathic Gaucher's Disease

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What is being collected

Data Collection

Who is being recruted

Mental Disorders+22

+ Brain Diseases

+ Brain Diseases, Metabolic

+3 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Observational
Study Start: September 1991
See protocol details

Summary

Principal SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Last updated: July 2, 2017
Sourced from a government-validated database.Claim as a partner

Study start date: September 23, 1991

Actual date on which the first participant was enrolled.

The purpose of this study is to examine the effects of enzyme replacement therapy in patients with neuronopathic Gaucher's disease and to investigate the pathogenesis of their neurological signs and symptoms. Macrophage-targeted glucocerebrosidase will be administered by intravenous infusion under the supervision of the patient's private physician at an initial dosage of 60 to 120 IU per kg of body weight weekly or every other week. Patients will be categorized as treatment responders if they display a clinically significant increase in hemoglobin concentration and a reduction in hepatic or splenic volume. Improvement in these parameters over time will be correlated with measurements for metabolic encephalopathy and radiologic, electrophysiologic and psychometric measurements of neurological involvement.

NCT00001289
Principal SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Last updated: July 2, 2017
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

70 patients to be enrolled

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

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.

Healthy volunteers not allowed

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

Conditions

Pathology

Mental DisordersBrain DiseasesBrain Diseases, MetabolicCentral Nervous System DiseasesLipid Metabolism, Inborn ErrorsLipidosesMetabolic DiseasesMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNeurologic ManifestationsNutritional and Metabolic DiseasesSigns and SymptomsSphingolipidosesPathological Conditions, Signs and SymptomsLysosomal Storage DiseasesNeurobehavioral ManifestationsLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornGenetic Diseases, InbornLipid Metabolism DisordersNeurodevelopmental DisordersGaucher DiseaseIntellectual DisabilitySeizures

Criteria

3 exclusion criteria prevent from participating
Patient and/or the patient's parent(s) or legal guardian(s) are unable to understand the nature, scope, and possible consequences of the study
Patient is unable to comply with the protocol, e.g., uncooperative with protocol schedule, refusal to agree to all of the study procedures
Patient who participates in a clinical study of an investigational therapeutic agent for Gaucher 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

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, United StatesOpen National Institutes of Health Clinical Center, 9000 Rockville Pike in Google Maps
CompletedOne Study Center