Phase 2 N=42 Treatment

Antithymocyte Globulin and Cyclosporine to Treat Myelodysplasia

Myelodysplastic Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT00005937 ↗

Enrolled (actual)

Serious AEs

26.2%

Results posted

Oct 2014

Primary outcome: Primary: Red Blood Cell Transfusion Independence — 6; 10; 21 participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Antithymocyte globulin (Drug); Cyclosporine (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Neal Young, M.D.
Primary completion: Mar 2008

Outcome Measures

Outcome	Result	p-value
PRIMARY Red Blood Cell Transfusion Independence	6; 10; 21	—

Summary

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: evaluate whether this drug combination can increase blood counts in patients and reduce their need for transfusions; compare survival of patients who respond to ATG and cyclosporine treatment with those who do not respond; and 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).

Eligibility Criteria

INCLUSION CRITERIA

MDS of refractory anemia (RA), refractory anemia with ring sideroblasts (RARS) & refractory anemia with excess blasts (RAEB) sub-types
Off all other treatments (except G-CSF (granulocyte colony stimulating factor), 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.
Eastern Cooperative Oncology Group (ECOG) performance status of 2 or less
High or intermediate predicted probability of response

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-CSF) 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00005937). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.