A Randomized Trial of Procrit vs. No Procrit in AML and High Risk MDS

Study Overview
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Brief Summary
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The goal of this clinical research study is to find out if Procrit (epoetin alfa) will help decrease the need for blood transfusions in patients who have Acute Myelogenous Leukemia (AML) or High-risk Myelodysplastic Syndrome (MDS) and are receiving chemotherapy. Researchers also want to learn about the remission rates (rates of recovery) in patients with cancer who have received treatment with epoetin alfa. The safety and effectiveness of this therapy will also be studied.

Detailed Description
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Epoetin alfa is a medication that helps the body make more red blood cells. Researchers want to find out if it will be effective in reducing the need for blood transfusions in patients who have AML or high-risk MDS and are receiving chemotherapy. If you are found to be eligible to take part in this study, you will be randomly assigned (as in the toss of a coin) to one of 2 treatment groups. Participants in one group will be given epoetin alfa along with blood transfusions, if the doctor thinks it is necessary. Participants in the other group will not receive epoetin alfa. Instead they will only have blood transfusions, which is the standard of care. No matter what group you are in, you will receive transfusions if your hemoglobin (an element of red blood cells that carries oxygen) drops below a certain level or if the doctor thinks it is necessary. You will be asked to keep a diary listing the dates of all transfusions you receive. The study doctor will monitor your hemoglobin levels by checking your standard blood tests done by your treating doctor. If your hemoglobin rises above a certain level, treatment with epoetin alfa may be temporarily stopped until your hemoglobin level decreases. If you are assigned to receive epoetin alfa, you will receive it once a week by subcutaneous (just under the skin) injection during your regularly scheduled chemotherapy. You will receive treatment with epoetin alfa for up to 12 weeks. If you experience any intolerable side effects or the disease gets worse, you will be taken off this study. Participants in both groups will continue to receive chemotherapy during this study as regularly scheduled. During chemotherapy (as part of your standard of care), you will have around 1 tablespoon of blood drawn every 1-2 weeks for routine blood tests. This is an investigational study. Epoetin alfa is FDA approved and commercially available. Up to 54 patients will take part in this study. All will be enrolled at the University of Texas (UT) MD Anderson Cancer Center.

Official Title
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Randomized Study of Procrit vs no Procrit in Patients With Newly Diagnosed Acute Myelogenous Leukemia (AML) or High-risk Myelodysplastic Syndrome (MDS) Undergoing Frontline Myelosuppressive Induction/Consolidation Chemotherapy

Conditions
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Acute Myelogenous Leukemia, Myelodysplastic Syndrome, Leukemia

Intervention / Treatment
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* Drug: Procrit


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with a diagnosis of AML or high-risk MDS (based on International Prognostic Scoring System (IPSS): refractory anemia with excess of blasts (RAEB) or RAEB in transformation [RAEB-t]) receiving frontline induction chemotherapy with any high dose or conventional dose cytarabine-containing regimen or clofarabine-containing regimen at MD Anderson Cancer Center. Patients must be enrolled on the study within two weeks of the start of induction chemotherapy. Patients with documented iron, vitamin B12, or folate deficiency are eligible, but should receive replacement therapy while on study. Understand and voluntarily sign an informed consent form. Exclusion Criteria: Patients with prior treatment with any form of erythropoietin within the previous month. Patients with uncontrolled hypertension (> or =140/90), uncontrolled, clinically significant cardiac arrhythmias, or history of pulmonary embolism or thrombosis within the last 5 years. New onset (within 3 months prior to randomization) or poorly controlled seizures. Patients with known hypersensitivity to the active substance or any of the excipients. Pregnant or lactating women. Acute Erythroleukemia (M6 French-American-British (FAB) classification) Hemoglobin greater than or equal to 10g/dl Patients with head and neck cancer receiving radiation therapy when erythropoiesis-stimulating agents (ESAs) were given to maintain hemoglobin levels of more than 12 g/dL. Patients with metastatic breast cancer receiving chemotherapy when ESAs were given to maintain hemoglobin levels of more than 12 g/dL. Patients with chronic kidney failure when ESAs were given to maintain hemoglobin levels of more than 12 g/dL. Patients requiring major surgery would be taken off study due to a higher chance of blood clots being reported while taking ESAs.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Supportive Care
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Procrit Arm<br>Participants receive Procrit along with blood transfusions. Procrit 40,000 units subcutaneously every week starting within two weeks (before or after) from the start of induction chemotherapy. | Drug: Procrit<br>* 40,000 units sq every week starting within two weeks (before or after) from the start of induction chemotherapy.<br>* Other names: Epogen;|
| No Intervention: No Procrit: Standard Arm<br>Participants do not receive Procrit before receiving blood transfusions. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Median Number of Participant Transfusions Required During 12 Weeks of Treatment | The number and frequency of packed red blood cells (PRBC) transfusions assessed and compared between two groups, treatment group (Procrit) and standard care group (No Procrit). Participants log all PRBC transfusions. Reported are the number of transfusions in the treatment arm during induction and consolidation chemotherapy with the concomitant use of epoetin alfa during therapy, and in the standard arm those that occured during same 12 week period. | 12 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Complete Remission | International Working Group (IWG) criteria for responses defined as: Complete Remission (CR) - Disappearance of all clinical and/or radiologic evidence of disease. Neutrophil count > 1.0 x 10^9/L and platelet count > 100 x 10^9/L, and normal bone marrow differential (< 5% blasts); Partial remission (PR): as CR except for presence of 5-25% marrow blasts and with a decrease of marrow blast at least 50%. | After 1 course of therapy, one course is 4 weeks. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Acute Myelogenous Leukemia, AML, Myelodysplastic Syndrome, MDS, Leukemia, Procrit, Epoetin Alfa, Epogen