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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 is a Phase 1 study that has completed, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 22 participants.

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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.

Interventions

  • DRUG Cyclophosphamide
  • DRUG Cyclosporine

Trial Details

FieldValue
Enrollment Target 22 participants
Start Date 2010-08
Est. Completion 2014-09
Phase Phase 1

What the Registry Record Tells You About NCT01193283

The ClinicalTrials.gov registry entry for NCT01193283 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 22 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 Cyclophosphamide is the first listed.

NCT01193283 reports 0 study locations.

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.

Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

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.