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NCT00152113 · ClinicalTrials.gov registry record · Phase 1
Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound
A Phase 1 study, sponsored by St. Jude Children's Research Hospital.
- Completed
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
NCT00152113: Completed Phase 1 study, sponsored by St. Jude Children's Research Hospital.
NCT00152113 is a Phase 1 study that has completed, run by St. Jude Children's Research Hospital. 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
NCT00152113, a Phase 1 study, has completed, sponsored by St. Jude Children's Research Hospital.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
Study Summary
Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body. Children may develop severe, debilitating clinical states, with stroke or abnormal blood flow to the brain. Treatment generally includes chronic blood transfusions which may cause iron overload, potentially leading to severe and sometimes fatal complications. Hematopoietic stem cell transplant using cells obtained from a sibling or an unrelated volunteer donor who is a perfect HLA "match" (same tissue type) for the recipient has shown to help, and possibly cure, sickle cell disease. Unfortunately, only about 10-20% of sickle cell patients have a HLA matched sibling donor, and the likelihood of locating an appropriate HLA matched unrelated donor through the various donor registries is limited. Stem cells from partially HLA matched family members (also called haploidentical transplant) is an option currently being explored for this patient population. This type of transplant has been used and found to be successful in some patients, mostly those with cancers of the blood. However, there can be significant complications with haploidentical transplant, primarily infection, failure of the graft to grow (graft failure), and a disorder called graft-versus-host disease. In addition, few patients with sickle cell disease have undergone this procedure. Therefore, the risks and benefits of haploidentical transplants for patients with sickle cell disorder are not as well established as those using an HLA identical sibling or unrelated donor. The primary objective of this study is to assess the safety of haploidentical stem cell transplantation for children and adolescents with severe sickle cell disease and stroke or abnormal transcranial Doppler ultrasound requiring chronic transfusion therapy. The treatment plan will be considered safe if there is not excessive toxicity. Toxicity for this protocol is defined as graft failure/graft rejection, severe acute GVHD
Interventions
- DEVICE CliniMACS
- PROCEDURE Hematopoietic Stem Cell Transplantation
- DRUG See intervention description
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2005-04 |
| Est. Completion | 2009-01 |
| Phase | Phase 1 |
What the finished NCT00152113 record still lists
NCT00152113 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 3 interventions - of which CliniMACS is the first listed.
NCT00152113 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00152113 about?
NCT00152113 is a clinical study titled "Haploidentical Stem Cell Transplant for Patients With Sickle Cell Disease and Prior Stroke or Abnormal Transcranial Ultrasound". Sickle cell disease is a life-long blood condition that can cause damage to the brain and other organs of the body. Children may develop severe, debilitating clinical states, with stroke or abnormal blood flow to the brain. Treatment generally includes chronic blood transfusions which may cause iron...
What is the current status of trial NCT00152113?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2005-04. Estimated completion is 2009-01.
What interventions are being tested in trial NCT00152113?
The interventions under investigation include: CliniMACS (DEVICE), Hematopoietic Stem Cell Transplantation (PROCEDURE), See intervention description (DRUG).
Who is sponsoring clinical trial NCT00152113?
This trial is sponsored by St. Jude Children's Research Hospital, which has 410 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00152113'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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