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NCT01410825 · ClinicalTrials.gov registry record · Phase 1
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
A Phase 1 study, sponsored by David Williams.
- Completed
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT01410825: Completed Phase 1 study, sponsored by David Williams.
NCT01410825 is a Phase 1 study that has completed, run by David Williams. 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
NCT01410825, a Phase 1 study, has completed, sponsored by David Williams.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal people have blood cells called platelets that stop bleeding when blood vessels are damaged. Boys with WAS have low numbers of platelets that do not function correctly. Boys with WAS are thus at risk for severe life-threatening bleeding. A normal immune system is made of special blood cells called white blood cells, which protect against infection and also fight certain types of cancer. In WAS, these white blood cells don't work as well as they should, making these boys very susceptible to infections and to a form of blood cancer known as lymphoma. The abnormal white blood cells of patients with WAS also cause diseases such as eczema and arthritis. Although WAS can be mild, severe forms need treatment as early as possible to prevent life-threatening complications due to bleeding, infection and blood cancer. Over the past decade, investigators have developed new treatments based on the investigators knowledge of the defective gene causing WAS. The investigators can now use genes as a type of medicine that will correct the problem in the patient's own bone marrow. The investigators call this process gene transfer. The procedure is very similar to a normal bone marrow transplant, in that the old marrow is killed off using chemotherapy, but is different because the patient's own bone marrow is given back after it is treated by gene transfer. This approach can be used even if the patient does not have any matched donors available and will avoid problems such as GVHD and rejection. The investigators wish to test whether this approach is safe and whether gene transfer will lead to the development of a healthy immune and blood system.
Primary Outcome
Safety of infusion of transduced cells as rescue of hematopoiesis after conditioning (hematopoietic recovery as assessed by absolute neutrophil count (ANC) above 0.5 x 109 /l for three consecutive days, achieved within 6 weeks following infusion).
Interventions
- BIOLOGICAL Retrovirus-mediated gene transfer
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2011-07 |
| Est. Completion | 2024-09 |
| Phase | Phase 1 |
What the finished NCT01410825 record still lists
NCT01410825 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 Retrovirus-mediated gene transfer is the first listed.
NCT01410825 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01410825 about?
NCT01410825 is a clinical study titled "Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome". The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome. Normal people have blood cells called platelets that stop bleeding when blood vessels are damaged. Boys with W...
What is the current status of trial NCT01410825?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2011-07. Estimated completion is 2024-09.
What interventions are being tested in trial NCT01410825?
The interventions under investigation include: Retrovirus-mediated gene transfer (BIOLOGICAL).
Who is sponsoring clinical trial NCT01410825?
This trial is sponsored by David Williams, which has 5 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01410825'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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