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NCT00061360 · ClinicalTrials.gov registry record · Phase 2
Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia
A Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 2
- Development phase
- 77
- Enrollment target
NCT00061360: Completed Phase 2 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT00061360 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 77 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (42% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00061360, a Phase 2 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 77 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 and immunosuppressive treatment with anti-thymocyte globulin (ATG) and cyclosporine (CsA) have dramatically changed the natural course of this illness, with 5 year survival of 75% in patients undergoing either treatment. Since most patients are not suitable candidates for hematopoietic stem cell transplantation (HSCT) due to advanced age or lack of a histocompatible sibling, efforts at NHLBI have focused on improving immunosuppression treatment in order to improve response rates, survival, and to decrease relapse. In our experience of 122 patients treated at NHLBI with the combination of ATG and cyclosporine, one quarter to one third did not respond; about 50% of responders relapsed; and 5 year survival was correlated with the robustness in blood cell count improvement at 3 months (reticulocyte or platelet count greater than or equal to 50,000 /uL). Why some patients do not respond initially while others relapse is unclear. Autoreactive T cells may be resistant to the effect of ATG/CsA (nonresponders), while in others residual autoreactive T cells expand post-treatment leading to hematopoietic stem cell destruction and recurrent pancytopenia (relapse). Therefore, novel immunosuppressive regimens to increase response rates and hematologic recovery at 3 months and to decrease relapse rates are needed. An ongoing NHLBI trial, which is close to completing accrual, has added mycophenolate mofetil (MMF) for a total of 18 months to standard ATG + CsA in an attempt to reduce the relapse rate after cyclosporine is discontinued. Preliminary results have been disappointing, with no marked reduction in relapse among patients who received MMF. Sirolimus (rapamycin, Rapamune , RAPA) is a novel immunosuppressive agent, which acts synergistically with cyclosporine by blocking T cell activation through
Primary Outcome
Up to 10/11/2005- Response rate at 3 months defined as no longer meeting criteria for SAAFrom 10/11/2005-relapse rates after initial standard treatment with ATG followed by CsA for six months
Interventions
- DRUG ATG+Rapamune+cyclosporine
- DRUG ATG+cyclosporine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 77 participants |
| Start Date | 2003-06-26 |
| Est. Completion | 2015-09-08 |
| Phase | Phase 2 |
What the finished NCT00061360 record still lists
NCT00061360 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 77 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (42% lower).
The record links to 0 conditions, and to 2 interventions - of which ATG+Rapamune+cyclosporine is the first listed.
NCT00061360 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT00061360 about?
NCT00061360 is a clinical study titled "Improving Immunosuppressive Treatment for Patients With 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 and immunosuppressive treatment with anti-thymocyte globulin (ATG) and cyclosporine (CsA) have dramatically changed the...
What is the current status of trial NCT00061360?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 77 participants. The study started on 2003-06-26. Estimated completion is 2015-09-08.
What interventions are being tested in trial NCT00061360?
The interventions under investigation include: ATG+Rapamune+cyclosporine (DRUG), ATG+cyclosporine (DRUG).
Who is sponsoring clinical trial NCT00061360?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT00061360's enrollment target sits among peer trials
77 891st of 2000 higher than 1,108 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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