Phase II Study of Horse Anti-Thymocyte Globulin (hATG), Cyclosporine, Methylprednisolone, and GCSF (Filgrastim or Pegfilgrastim) in Patients with Aplastic Anemia (AA), or Low/Int-1 Risk Myelodysplastic Syndrome (MDS)
Antithymocyte Globulin,CYCLOSPORIN A,G-CSF,Pegfilgrastim
If you are reading and signing this form on behalf of a potential participant, please note: Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant. You are being asked to take part in this study because you have aplastic anemia (AA) or low/int-1 risk myelodysplastic syndrome (MDS). The goal of this clinical research study is to learn if horse anti-thymocyte globulin (hATG), given in combination with methylprednisolone, cyclosporine, and G-CSF (filgrastim or pegfilgrastim), can help to control AA and/or low-int-1 risk MDS. The safety of this drug combination will also be studied. hATG is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in AA and in some cases of MDS, killing these cells may help treat the disease. Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in AA and in some cases of MDS. Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count.
Disease Group: Leukemia
Treatment Agent: Antithymocyte Globulin,CYCLOSPORIN A,G-CSF,Pegfilgrastim
Treatment Location: Only at MDACC
Primary Objective To evaluate the efficacy of the combination of hATG, methylprednisolone, cyclosporine, and GCSF in achieving response (complete response [CR], partial response [PR], or hematologic improvement [HI]) in patients with Aplastic Anemia, or MDS. Secondary Objectives To assess the safety, tolerability, and toxicities of the combination of hATG, methylprednisolone, cyclosporine, and GCSF in patients with Aplastic Anemia, or MDS. To assess time to response, response duration, and overall survival of patients with Aplastic Anemia, or MDS being treated with the combination of hATG, methylprednisolone, cyclosporine, and GCSF.
IRB Review and Approval Date: 06/25/2012
Recruitment Status: Open
Projected Accrual: N/A
1) Patients with the diagnosis of MDS (Low, Int-1 by IPSS, or
hypocellular) who are either previously treated or untreated are
eligible for this trial.
2) Patients with the diagnosis of aplastic anemia who are either previously treated or untreated are eligible if they are not currently candidates for an allogeneic stem cell transplant.
3) All ages are eligible.
4) Patients must have been off of cytotoxic, immunosuppressive (except steroids), or targeted therapy for at least 2 weeks prior to entering this study, and have recovered from the toxic effects of that therapy to grade 1 or less.
5) Adequate organ function as defined as: liver function (bilirubin < 2mg/dL, AST <3 x ULN), kidney function (creatinine < 2.5 x ULN ).
6) ECOG performance status of </= 2.
7) Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately.
8) A negative urine pregnancy test is required within 1 week for all women of childbearing potential prior to enrolling on this trial.
9) Patient must have the ability to understand the requirements of the study and signed informed consent. A signed informed consent by the patient or his legally authorized representative is required prior to their enrollment on the protocol.
10) Patients should have an indication for therapy for their disease such as transfusion dependence or morbidity associated with their cytopenia(s) such as bleeding, severe fatigue, or frequent/multiple infections (eg. neutropenia).
1) Pregnant women are excluded from this study. Because there is an
unknown but potential risk for adverse events in nursing infants
secondary to treatment of the mother with the study agents,
breastfeeding should be discontinued if the mother is treated on this study.
2) Known HIV infection
3) Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
4) Patient with documented hypersensitivity to any of the component medications.
Information and next steps
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