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NCT01193283 · ClinicalTrials.gov registry record · Phase 1
Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia
A Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Completed
- Registry status
- Phase 1
- Development phase
- 22
- Enrollment target
NCT01193283: Completed Phase 1 study, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT01193283 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 22 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01193283, a Phase 1 study, has completed, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 22 participants
- Enrollment target
Study Summary
Background: * Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard treatment for SAA involves injections of antithymocyte globulin (ATG) plus cyclosporine (CsA). This regimen has been shown to improve the blood counts in about two-thirds of patients. However, the ATG/CsA regimen has the following limitations: (a) the disease can come back (relapse) in about one-third of patients who improve initially; and (b) in about 10% to 15% of cases, certain types of bone marrow cancer (such as myelodysplasia and leukemia) can develop (called evolution). Experience with other drugs in SAA such as cyclophosphamide suggests that similar response rates to ATG/CsA can be achieved with a lower risk of relapse and clonal evolution. However, cyclophosphamide was found to have significant side effects in SAA when investigated over 10 years ago due to increase risk of fungal infections. * Better antibiotic drugs against fungus have been developed and are widely used to treat patients who have low white blood cell counts and are at risk of developing infections. In SAA patients in particular, these newer antibiotics have had a large impact in preventing and treating fungus infections. Researchers are revisiting the use of cyclophosphamide in SAA treatment, and plan to give a lower dose of CsA in combination with the immune-suppressing drug cyclophosphamide, as well as antibiotics to protect against infections, as a possible treatment for the disease. Objectives: \- To determine the safety and effectiveness of the combination of cyclophosphamide and cyclosporine in treating severe aplastic anemia that has not been treated with immunosuppressive therapy.
Primary Outcome
The safety endpoint will be toxicity profile after 6 months of treatment. The efficacy endpoint is complete response rate at 6 months, with complete response defined as blood counts no longer meeting the standard criteria for severe pancytopenia in severe aplastic anemia.
Interventions
- DRUG Cyclophosphamide
- DRUG Cyclosporine
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2010-08 |
| Est. Completion | 2014-09 |
| Phase | Phase 1 |
What the finished NCT01193283 record still lists
NCT01193283 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 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (63% lower).
The record links to 0 conditions, and to 2 interventions - of which Cyclophosphamide is the first listed.
NCT01193283 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT01193283 about?
NCT01193283 is a clinical study titled "Cyclophosphamide Plus Cyclosporine in Treatment-Naive Severe Aplastic Anemia". Background: * Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions. Standard treatment for SAA involves injections of antithymocyte globulin (ATG) plus cyclosporine (CsA). This regimen has been shown to im...
What is the current status of trial NCT01193283?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 22 participants. The study started on 2010-08. Estimated completion is 2014-09.
What interventions are being tested in trial NCT01193283?
The interventions under investigation include: Cyclophosphamide (DRUG), Cyclosporine (DRUG).
Who is sponsoring clinical trial NCT01193283?
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 NCT01193283's enrollment target sits among peer trials
22 1601st of 2000 higher than 386 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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