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NCT00001234 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
A Phase 1 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- Completed
- Registry status
- Phase 1
- Development phase
- 120
- Enrollment target
NCT00001234: Completed Phase 1 study, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
NCT00001234 is a Phase 1 study that has completed, run by National Institute of Neurological Disorders and Stroke (NINDS). The registered enrollment target is 120 participants, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (100% higher). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001234, a Phase 1 study, has completed, sponsored by National Institute of Neurological Disorders and Stroke (NINDS).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 120 participants
- Enrollment target
Study Summary
Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages. The most severe form, Type 2 Gaucher's Disease occurs in infants who die in the first years of life (with rapidly progressive neurologic deterioration). The condition is passed from generation to generation through autosomal recessive inheritance. Fabry's disease isa genetic disorder (X-linked recessive) due to the absence of the enzyme a-galactosidase A. The disease is characterized by abnormal collections of glycolipids in cells (histiocytes) within blood vessel walls, tumors on the thighs, buttocks, and genitalia(2) decreased sweating, tingling sensations in the extremities, and cataracts. Patients with Fabry's disease die from complications of the kidney, heart, or brain. Both conditions are caused by the absence of specific enzymes (3). Patients with these conditions are missing (3) or have defective genes needed for the normal production of these enzymes. Studies on the blood-forming cells in bone marrow have lead to gene therapies using retroviruses as vehicles to carry and insert working genes into abnormal or diseased cells. This study is designed to measure the safety and effectiveness of transferring working copies of genes responsible for making missing enzymes into the cells of patients with Gaucher's or Fabry disease.
Interventions
- GENETIC human glucocerebrosidase cDNA
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 120 participants |
| Start Date | 1988-01 |
| Est. Completion | 2002-04 |
| Phase | Phase 1 |
What the finished NCT00001234 record still lists
NCT00001234 is an interventional study that assigns participants to a tested intervention. The registered 120 participants enrollment target is mid-sized for trials with a published cap, above the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (100% higher).
The record links to 0 conditions, and to 1 intervention - of which human glucocerebrosidase cDNA is the first listed.
NCT00001234 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00001234 about?
NCT00001234 is a clinical study titled "Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells". Gaucher's disease is a lysosomal storage disease resulting from glycocerebroside GLUCOCEREBROSIDE (1) accumulation in macrophages due to a genetic deficiency of the enzyme glucocerebrosidase. It may occur in patients of all ages. The most severe form, Type 2 Gaucher's Disease occurs in infants who d...
What is the current status of trial NCT00001234?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 120 participants. The study started on 1988-01. Estimated completion is 2002-04.
What interventions are being tested in trial NCT00001234?
The interventions under investigation include: human glucocerebrosidase cDNA (GENETIC).
Who is sponsoring clinical trial NCT00001234?
This trial is sponsored by National Institute of Neurological Disorders and Stroke (NINDS), which has 567 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00001234's enrollment target sits among peer trials
120 479th of 2000 higher than 1,503 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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