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NCT01565616 · ClinicalTrials.gov registry record · Phase 2
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
A Phase 2 study, sponsored by Emory University.
- Completed
- Registry status
- Phase 2
- Development phase
- 22
- Enrollment target
NCT01565616: Completed Phase 2 study, sponsored by Emory University.
NCT01565616 is a Phase 2 study that has completed, run by Emory University. The registered enrollment target is 22 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01565616, a Phase 2 study, has completed, sponsored by Emory University.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 22 participants
- Enrollment target
Study Summary
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features. The primary goal of this multi-center Phase II study is to determine the safety and feasibility of a conditioning regimen consisting of busulfan (Bu)/ fludarabine (Flu)/ anti-thymocyte globulin (ATG) in adult patients with severe SCD. A two-component design will be used for this study. The first component will be restricted to patients who have an HLA-identical sibling donor. Five patients will be transplanted during the first component of the study. If no more than 2 of the first 5 patients experience unacceptable toxicity, including death, within the first six months after transplantation, then the safety of the regimen will be considered promising in adult SCD patients. The second component will include patients who have a related or an unrelated human leukocyte antigen (HLA) matched donor. Up to 15 additional patients will be transplanted in this component of the study which will evaluate the safety and feasibility of unrelated donor hematopoietic cell transplantation (HCT) in adults with SCD. Data related to study endpoints for 1 year after transplantation will be collected; however, participating centers will be encouraged to conduct long-term follow-up evaluations of patients according to standard institutional guidelines. The purpose of this pilot safety trial is to see if this approach is feasible and meets accrual goals lending support to the development of a subsequent full scale investigation of HCT and comparing outcomes in a transplantation cohort to a control cohort of adults eligible for, but unwilling or unable to receive HCT treated by supportive therapy with a primary endpoint of five years survival for this full scale comparative trial.
Primary Outcome
Event-free survival is defined as stable donor erythropoiesis with no new clinical evidence of sickle cell disease. Primary or late graft rejection, disease recurrence, and death are considered events for this endpoint.
Interventions
- DRUG Conditioning Regimen with Bone Marrow Transplant
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2012-03 |
| Est. Completion | 2016-06-30 |
| Phase | Phase 2 |
What the finished NCT01565616 record still lists
NCT01565616 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 22 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 1 intervention - of which Conditioning Regimen with Bone Marrow Transplant is the first listed.
NCT01565616 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01565616 about?
NCT01565616 is a clinical study titled "Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease". This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features. The primary goal of this multi-center Phase II study is to determine the...
What is the current status of trial NCT01565616?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 22 participants. The study started on 2012-03. Estimated completion is 2016-06-30.
What interventions are being tested in trial NCT01565616?
The interventions under investigation include: Conditioning Regimen with Bone Marrow Transplant (DRUG).
Who is sponsoring clinical trial NCT01565616?
This trial is sponsored by Emory University, which has 1,208 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT01565616's enrollment target sits among peer trials
22 1790th of 2000 higher than 207 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 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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