Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 is a Phase 2 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 77 participants.
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
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00061360
The ClinicalTrials.gov registry entry for NCT00061360 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 77 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 2 interventions - of which ATG+Rapamune+cyclosporine is the first listed.
NCT00061360 reports 0 study locations.
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
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.