Completed

Retroviral-Mediated Transfer and Expression of Glucocerebrosidase and Ceramidtrihexosidase (a-Galactosidase A) cDNA's in Human Hematopoietic Progenitor Cells

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What is being tested

human glucocerebrosidase cDNA

Genetic
Who is being recruted

Brain Diseases+14

+ Brain Diseases, Metabolic

+ Central Nervous System Diseases

See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1
Interventional
Study Start: January 1988
See protocol details

Summary

Principal SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Last updated: March 4, 2008
Sourced from a government-validated database.Claim as a partner

Study start date: January 1, 1988

Actual date on which the first participant was enrolled.

This protocol was developed in order to obtain bone marrow stem cells for ex vivo transduction with retroviruses containing the human glucocerebrosidase gene. We continue to enter a small number of patients to this protocol each year. Studies with the bone marrow hematopoietic progenitor cells have enabled us to identify the most effective retroviral construct currently available in order to carry out gene therapy trials in patients with Gaucher's disease. The data revealed that a comparatively simple retroviral construct containing human glucocerebrosidase cDNA driven by the MoLV promoter is highly effective. We have obtained approval and initiated a Phase I safety and gene marking investigation in patients with Type I Gaucher's Disease.

NCT00001234
Principal SponsorNational Institute of Neurological Disorders and Stroke (NINDS)
Last updated: March 4, 2008
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

120 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.

Healthy volunteers 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 DiseasesMetabolism, Inborn ErrorsCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesNutritional and Metabolic DiseasesSphingolipidosesLysosomal Storage DiseasesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornGenetic Diseases, InbornLipid Metabolism DisordersGaucher Disease

Criteria

Normal and patient volunteers. Individuals with platelet counts less than 40,000/ul, PT greater than 15 seconds, or PTT greater than 40 seconds will not undergo bone marrow aspiration. Individuals with hematologic disorders other than Gaucher Disease, Fabry Disease, or mild iron deficiency will not undergo bone marrow aspiration. HIV positive individuals will be excluded from participating.

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 Institute of Neurological Disorders and Stroke (NINDS)

Bethesda, United StatesOpen National Institute of Neurological Disorders and Stroke (NINDS) in Google Maps
CompletedOne Study Center