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NCT00001735 · ClinicalTrials.gov registry record · Phase 1
Gene Therapy for Gyrate Atrophy
A Phase 1 study, sponsored by National Eye Institute (NEI).
- Completed
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT00001735: Completed Phase 1 study, sponsored by National Eye Institute (NEI).
NCT00001735 is a Phase 1 study that has completed, run by National Eye Institute (NEI). The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00001735, a Phase 1 study, has completed, sponsored by National Eye Institute (NEI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
This study will evaluate the safety and effectiveness of gene therapy for patients with gyrate atrophy, an inherited condition in which areas of the retina-the inner lining of the wall of the eye-become thin. Over several decades, this degeneration of the retina causes tunnel vision, night blindness, and other vision problems. Gyrate atrophy is caused by a defect in the gene responsible for producing an enzyme, ornithine aminotransferase (OAT), that breaks down an amino acid called ornithine. As a result, excessive ornithine buildup causes the retinal thinning. Currently, this condition can only be treated with amino acid tablets and a very low-protein diet with limited fruits and vegetables and more than 2,000 calories a day from carbohydrates and fats. Some patients cannot maintain this diet, and they need another treatment. One possible alternative is to replace the defective gene with one that functions normally. Patients who have been followed in NEI's Ocular Genetics service may be eligible to participate in this study. Study patients will undergo the following gene therapy procedure: 1. Skin biopsy-A small piece of skin is surgically removed from the patient's thigh. 2. Gene transfer-Skin cells called keratinocytes are taken from the biopsied tissue and grown in the laboratory. The normal gene that produces OAT is inserted into the cells, causing them to produce more of the enzyme. 3. Skin graft-Under local anesthesia, a patch of skin about 2 1/4 inches x 2 1/4 inches is surgically removed from the upper thigh and some of the cells with increased OAT are grafted back onto this area. Patients will be followed at 1 week and 2 weeks after the procedure, then monthly for 6 months, again at 9 months and 1 year. Follow-up will continue at 1-year intervals in patients in whom the treatment is successful. During each follow-up visit patients will have 2 to 3 tablespoons of blood drawn for tests. A small biopsy (about 1/4 inch) of transplanted cells will also be d
Interventions
- PROCEDURE Gene therapy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 1998-04 |
| Est. Completion | 2000-10 |
| Phase | Phase 1 |
What the finished NCT00001735 record still lists
NCT00001735 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).
The record links to 0 conditions, and to 1 intervention - of which Gene therapy is the first listed.
NCT00001735 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00001735 about?
NCT00001735 is a clinical study titled "Gene Therapy for Gyrate Atrophy". This study will evaluate the safety and effectiveness of gene therapy for patients with gyrate atrophy, an inherited condition in which areas of the retina-the inner lining of the wall of the eye-become thin. Over several decades, this degeneration of the retina causes tunnel vision, night blindness...
What is the current status of trial NCT00001735?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 1998-04. Estimated completion is 2000-10.
What interventions are being tested in trial NCT00001735?
The interventions under investigation include: Gene therapy (PROCEDURE).
Who is sponsoring clinical trial NCT00001735?
This trial is sponsored by National Eye Institute (NEI), which has 221 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00001735's enrollment target sits among peer trials
5 1989th of 2000 higher than 6 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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