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NCT00260689 · ClinicalTrials.gov registry record · Phase 2
Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 136
- Enrollment target
NCT00260689 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 136 participants.
The verdict
NCT00260689, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 136 participants
- Enrollment target
Study Summary
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Allogeneic bone marrow transplantation offers the opportunity for cure in 70% of patients, but most patients are not suitable candidates for hematopoietic stem cell transplantation (HSCT) due to advanced age or lack of a histocompatible donor. For these patients, comparable long term survival is attainable with immunosuppressive treatment with anti-thymocyte globulin (ATG) and cyclosporine (CsA). However, of those patients treated with horse ATG(h-ATG)/CsA, one quarter to one third will not respond, and about 50% of responders relapse. Auto-reactive T cells may be resistant to the effect of ATG/CsA (non-responders), while in others residual auto-reactive T cells expand post-treatment, leading to hematopoietic stem cell destruction and recurrent pancytopenia (relapse). As long term survival is correlated to response rates and robustness of hematopoietic recovery, novel immunosuppressive regimens that can achieve hematologic response and decrease relapse rates are needed. This trial will compare the effectiveness of three immunosuppressive regimens as first line therapies in patients with SAA with early hematologic response as the primary endpoint, as well as assess the role of extended CsA treatment after h-ATG in reducing numbers of late events of relapse and clonal evolution. Randomization is employed to obtain an equal distribution of subject to each arm; comparisons of early hematologic responses will be made among the rates observed among the three concurrent arms (rabbit-ATG \[r-ATG\] versus standard h-ATG; alemtuzumab vs standard h-ATG). For long course CSA, comparison of primary end points will be to well established historic relapse rate of 38% at 2-3 years and a cumulative rate of clonal evolution of 15%.
Interventions
- DRUG Alemtuzumab
- DRUG Cyclosporine
- BIOLOGICAL Anti-thymocyte globulin (rabbit)
- BIOLOGICAL Anti-thymocyte globulin (horse)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 136 participants |
| Start Date | 2005-11-28 |
| Est. Completion | 2016-05-04 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00260689
The ClinicalTrials.gov registry entry for NCT00260689 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 136 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 501 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 4 interventions - of which Alemtuzumab is the first listed.
NCT00260689 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00260689 about?
NCT00260689 is a clinical study titled "Three Immunosuppressive Treatment Regimens for Severe Aplastic Anemia". Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow. Allogeneic bone marrow transplantation offers the opportunity for cure in 70% of patients, but most patients are not suitable candidates for hematopoietic ste...
What is the current status of trial NCT00260689?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 136 participants. The study started on 2005-11-28. Estimated completion is 2016-05-04.
What interventions are being tested in trial NCT00260689?
The interventions under investigation include: Alemtuzumab (DRUG), Cyclosporine (DRUG), Anti-thymocyte globulin (rabbit) (BIOLOGICAL), Anti-thymocyte globulin (horse) (BIOLOGICAL).
Who is sponsoring clinical trial NCT00260689?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
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