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| Sponsored by: |
National Heart, Lung, and Blood Institute (NHLBI) |
| Information provided by: | National Institutes of Health Clinical Center (CC) |
| ClinicalTrials.gov Identifier: | NCT00005937 |
Purpose
This study will determine the safety and effectiveness of a combination of the immune-suppressing drugs antithymocyte globulin (ATG) and cyclosporine for treating myelodysplasia, a disorder of low blood cell counts. It will: 1) evaluate whether this drug combination can increase blood counts in patients and reduce their need for transfusions; 2) compare survival of patients who respond to ATG and cyclosporine treatment with those who do not respond; and 3) determine the side effects of the treatment.
Myelodysplasia is thought to result from an immune system abnormality in which cells called lymphocytes attack the marrow's blood-forming cells. The resulting deficiencies of platelets and red and white blood cells cause anemia, susceptibility to infections, and easy bruising and bleeding. Various therapies, such as blood transfusions for anemia and bleeding, antibiotics for infection, chemotherapy and bone marrow transplantation are used to treat myelodysplasia, but all have disadvantages and some carry serious risks.
Patients 18 years of age and older with myelodysplasia may be eligible for this study. Candidates will be screened with a physical examination and medical history, blood tests, chest X-ray, electrocardiogram and bone marrow biopsy (removal of a marrow sample from the hipbone for microscopic examination). For this procedure, the hip area is anesthetized and a special needle is used to draw marrow from the bone.
Participants will be admitted to the NIH Clinical Center for the first 10 days of treatment and will then continue therapy on an outpatient basis. They will undergo the following tests and procedures:
During hospitalization, blood will be drawn daily for blood counts and other tests. Upon the patient's discharge after 10 days, the referring physician will do blood tests weekly during the first month of treatment and then every 2 weeks for the rest of the time the patient is taking cyclosporine. Dosages of this drug may be adjusted depending on the test results. Patients will be evaluated at the NIH Clinical Center at 3-month intervals for the first year, then every 6 months for the next 3 years and then at yearly intervals. A blood sample will be drawn at each visit. Bone marrow biopsies will be done at 6-month intervals for the first 3 years after treatment.
| Condition | Intervention | Phase |
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Myelodysplastic Syndrome |
Drug: Antithymocyte globulin Drug: Cyclosporine |
Phase II |
| MedlinePlus related topics: | Blood Transfusion and Donation |
| ChemIDplus related topics: | Cyclosporine Cyclosporin |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | A Phase II Study of Antithymocyte Globulin (ATG) and Cyclosporine to Treat the Cytopenia of Myelodysplastic Syndrome (MDS) |
| Estimated Enrollment: | 42 |
| Study Start Date: | June 2000 |
A growing body of laboratory and clinical evidence suggests that the cytopenia of MDS is at least partly a result of cytotoxic T cell activity. Treatments to abrogate T cell activity such as anti-thymocyte globulin alone and cyclosporine alone have demonstrated varying degrees of success in alleviating the cytopenia of MDS. A response to such therapy in MDS is associated with improved survival. Experience with aplastic anemia suggests that the combination of these two agents should be more effective in suppressing cytotoxic T cell activity and alleviating cytopenia. This protocol proposes using the combination of antithymocyte globulin (ATG) and cyclosporine (CSA) to treat the cytopenia of MDS, in an effort to improve the response rate to immunosuppressive therapy in this disease.
Eligibility
| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
MDS of RA, RARS & RAEB sub-types
Off all other treatments (except G-CSF, and transfusion support and related medications) for at least four weeks.
G-CSF can be used before, during and after the protocol treatment for patients with documented neutropenia (less than 500/uL) as long as they meet the criteria for anemia and/or thrombocytopenia as stated above.
ECOG performance status of 2 or less
EXCLUSION CRITERIA:
MDS of FAB sub-group chronic myelomonocytic leukemia (CMML)
Transformation to acute leukemia (FAB sub-group RAEB-T, ie., greater than 20% blasts in marrow aspirate)
Hypoplastic marrow without one major or two minor criteria
Treatment with growth factors (except for G-SCF) or cyclosporine within 4 weeks prior to entry to protocol
ECOG performance status of greater than 2
Active uncontrolled infection
Current pregnancy, or unwilling to take oral contraceptives if of childbearing potential
Patients for whom bone marrow transplant is indicated as standard therapy (age less than fifty-five with a fully-matched sibling donor)
Age less than18 years
Not able to give informed consent
HIV positive patients
Active malignant disease (excluding basal cell carcinoma)
Serum creatinine greater than 2mg/dl
Patients who are moribund or patients with concurrent hepatic, renal, cardiac, metabolic, or any disease of such severity that death within 3 months is likely
Low predicted probability of response
Contacts and Locations More Information
NIH Clinical Center Detailed Web Page
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| Responsible Party: | National Institutes of Health ( Elaine M. Sloand, M.D./National Heart, Lung, and Blood Institute ) |
| Study ID Numbers: | 000169, 00-H-0169 |
| First Received: | July 6, 2000 |
| Last Updated: | September 19, 2008 |
| ClinicalTrials.gov Identifier: | NCT00005937 |
| Health Authority: | United States: Federal Government |
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