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NCT01129544 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector
A Phase 1 study, sponsored by David Williams.
- Completed
- Registry status
- Phase 1
- Development phase
- 8
- Enrollment target
NCT01129544 is a Phase 1 study that has completed, run by David Williams. The registered enrollment target is 8 participants.
The verdict
NCT01129544, a Phase 1 study, has completed, sponsored by David Williams.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 8 participants
- Enrollment target
Study Summary
Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemotherapy, and graft versus host disease. The idea behind gene transfer is to replace the broken gene by putting a piece of genetic material (DNA) that has the normal gene into the child's cells. Gene transfer can only be done if we know which gene is missing or broken in the patient. For SCID-X1, gene transfer has been done in the laboratory and in two previous clinical trials by inserting the normal gene into stem cells from bone marrow. The bone marrow is the "factory" inside the bones that creates blood and immune cells. So fixing the gene in the bone marrow stem cells should fix the immune problem, without giving chemotherapy and without risk of graft versus host disease, because the child's own cells are used, rather than another person's. Out of the 20 subjects enrolled in the two previous trials, 18 are alive with better immune systems after gene transfer. Two of the surviving subjects received gene corrected cells over 10 years ago. Gene transfer is still research for two reasons. One is that not enough children have been studied to tell if the procedure is consistently successful. Of the 20 children enrolled in the previous two trials, one child did not have correction of the immune system, and died of complications after undergoing stem cell transplant. The second important reason why gene transfer is research is that we are still learning about the side effects of gene transfer and how to do gene transfer safely. In the last two trials, 5 children have experienced a serious side effect. These children developed leukemia related to the gene transfer itself. Leukemia is a cancer of the white blood cells, a condition where a few white blood cells grow out of control
Interventions
- BIOLOGICAL Gene transfer
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 8 participants |
| Start Date | 2010-04 |
| Est. Completion | 2023-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01129544
The ClinicalTrials.gov registry entry for NCT01129544 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 8 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is David Williams, which has 5 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Gene transfer is the first listed.
NCT01129544 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01129544 about?
NCT01129544 is a clinical study titled "Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector". Researchers are working on ways to treat SCID patients who don't have a matched brother or sister. One of the goals is to avoid the problems that happen with stem cell transplant from parents and unrelated people, such as repeat transplants, incomplete cure of the immune system, exposure to chemothe...
What is the current status of trial NCT01129544?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 8 participants. The study started on 2010-04. Estimated completion is 2023-03.
What interventions are being tested in trial NCT01129544?
The interventions under investigation include: Gene transfer (BIOLOGICAL).
Who is sponsoring clinical trial NCT01129544?
This trial is sponsored by David Williams, which has 5 total clinical trials registered on ClinicalTrials.gov.
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