October 2015 Vol 6, NO 5

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Clinical Trials Tracker

Trials Under Way for Patients with Myelodysplastic Syndrome

The following clinical trials are currently recruiting patients with myelodysplastic syndrome for inclusion in a variety of studies targeting a specific subpopulation of patients with this syndrome. Each trial description includes the NLM Identifier to use as a reference with ClinicalTrials.gov. The information below can help oncology nurse navigators direct their appropriate patients to a specific clinical trial described here.

1 Chemotherapy for Patients with Advanced Myelodysplastic Syndrome

The purpose of this open-label study is to examine the feasibility of initiating chemotherapy in patients with acute myeloid leukemia (AML) or with advanced myelodysplastic syndrome (MDS) in an outpatient setting, because introducing patients to chemotherapy in an outpatient setting may improve quality of life and lead to healthcare cost-savings. Patients aged ≥18 years with AML or with high-risk MDS (defined as 10%-19% blasts in marrow by morphology, flow cytometry, or blood), adequate cardiac function, and no sign of active viral, bacterial, or fungal infections may be eligible for enrollment if other criteria are met. Eligible patients will receive outpatient induction chemotherapy.

The primary outcome measures are the rate of hospital admissions during the first 4 to 7 days of outpatient chemotherapy, and the treatment-related mortality rate (measured during the first 14 days after chemotherapy induction). This study plans to enroll 25 patients in Washington and in Montana. For more information, contact Jack O. Hensold at 406-585-5070, or Pamela S. Becker at 206-616-1589. The NLM Identifier is NCT01807091.

2 BI 836858 for Patients with Myelodysplastic Syndrome

This open-label, parallel-assignment, phase 1/2 trial will investigate the maximum tolerated dose, safety and tolerability, pharmacokinetics, exploratory biomarker, and efficacy of BI 836858 as monotherapy and in addition to best supportive care in patients with myelodysplastic syndromes with symptomatic anemia. Patients aged ≥18 years with an Eastern Cooperative Oncology Group performance status ≤2 who have not received allogeneic bone marrow or stem-cell transplantation may be eligible to enroll if other criteria are met. Eligible patients will be randomized to receive best supportive care alone, or BI 836858 plus best supportive care.

The primary outcome measures of the phase 1 and phase 2 portions of the trial are the maximum tolerated dose and occurrence of dose-limiting toxicity, and the number of patients who receive red blood cell transfusions, respectively. The secondary outcome measures include the number of patients with neutrophil and platelet responses, overall objective response, and duration of response. This study expects to enroll 200 patients in Arizona, Florida, and Ohio. For more information, contact the Boehringer Ingelheim Call Center at 800-243-0127 or clintriage.rdg@boehringer-ingelheim.com. The NLM Identifier is NCT02240706.

3 Lenalidomide with/without Epoetin Alfa for Myelodysplastic Syndrome and Anemia

Investigators in this randomized, open-label, phase 3 clinical trial are comparing the efficacy of lenalidomide with or without epoetin alfa in patients with myelodysplastic syndrome (MDS) and anemia. Patients aged ≥18 years with a documented diagnosis of MDS lasting ≥3 months, marrow iron stores (ie, not iron deficient), and who have not had nontransfusion therapy for their MDS for 28 days before initiation of the trial treatment may be eligible for enrollment if other criteria are met. Eligible patients will be randomized to receive either lenalidomide monotherapy or lenalidomide in combination with epoetin alfa.

The primary outcome measures are the major erythroid response rate, defined as sustained transfusion independence in transfusion-dependent patients, or a rise in hemoglobin >2 g/dL in transfusion-independent patients with anemia for ≥8 consecutive weeks. Secondary outcome measures include the complete and partial bone marrow response rate, the major erythroid response rate, and the cytogenetic response rate. This study plans to enroll 252 patients at multiple locations across the United States. For more information, contact Peter D. Eisenberg at 415-925-5000 or info@marinspecialtycare.com, or Alan F. List at 813-745-7101 or alan.list@moffitt.org. The NLM Identifier is NCT00843882.

4 Azacitidine with/without Birinapant for Myelodysplastic Syndrome or Myelomonocytic Leukemia

This randomized, double-blind, phase 2 clinical trial will examine the use of azacitidine with or without birinapant in patients with high-risk myelodysplastic syndrome (MDS) or with chronic myelomonocytic leukemia. Patients aged ≥18 years with an Eastern Cooperative Oncology Group performance status 0, 1, or 2; adequate renal and liver function; and (in women) a negative serum pregnancy test at screening ≤96 hours before the first study dose may be eligible to enroll if other criteria are met. Eligible patients will be randomized to receive azacitidine with birinapant, or azacitidine with a placebo.

The primary outcome measure is the response rate, measured until disease progression for an expected average of 1 year. Secondary outcome measures include hematologic improvement, relapse-free survival, change in transfusion requirements, and an adverse events profile. This study expects to enroll 158 patients at multiple locations across the United States and abroad. For more information, contact AnnMarie Hulstine at 610-889-9900 (ext. 140) or annmarie.hulstine@tetralogicpharma.com, or Judy Schnyder at 610-889-9900 (ext. 151) or judy.schnyder@tetralogicpharma.com. The NLM Identifier is NCT02147873.

5 Associated Outcomes of Myelodysplasia Transplantation

Investigators in this observational, cohort study will compare overall survival in older adults with myelodysplastic syndrome (MDS) who do and do not receive reduced-intensity conditioning hematopoietic stem-cell transplantation (HSCT). Patients aged 60 to 75 years with an Eastern Cooperative Oncology Group performance status 0, 1, or 2; adequate organ function that permits reduced-intensity conditioning HSCT; and who are willing to undergo human leukocyte antigen typing and consider subsequent HSCT may be eligible to enroll if other criteria are met. Eligible patients will be split into cohorts that receive or do not receive HSCT.

The primary outcome measure is the overall survival, measured over a 5-year period. This study expects to enroll 290 patients in Boston, MA. For more information, contact Gregory A. Abel, MD, MPH, at 617-632-2304 or gaabel@partners.org, or Corey Watts at 617-582-7396 or coreyd_watts@dfci.harvard.edu. The NLM Identifier is NCT02390414.

6 Lenalidomide and Eltrombopag Olamine for Symptomatic Anemia in Myelodysplastic Syndrome

The purpose of this phase 2, nonrandomized, open-label study is to examine how well lenalidomide and eltrombopag olamine work together in treating patients with symptomatic anemia associated with low or intermediate myelodysplastic syndrome. Patients aged ≥18 years with an Eastern Cooperative Oncology Group performance status 0 to 3; International Prognostic Scoring System categories of low or intermediate-1–risk disease; and who do not have uncontrolled hypertension may be eligible for enrollment if other criteria are met. Eligible patients will receive lenalidomide and eltrombopag olamine in varying dosages specific to their treatment arm.

The primary outcome measures are the safety and tolerability of the treatment combination, as well as the rate of hematologic improvement as defined by the International Working Group criteria. Secondary outcome measures include bone marrow morphologic and cytogenetic responses, platelet counts, and bleeding events. This study expects to enroll 60 patients in Lawrence, KS; Rochester, MN; and Bronx, NY. For more information, contact Amit K. Verma at 718-430-8761 or amit.verma@einstein.yu.edu. The NLM Identifier is NCT01772420.

7 Mocetinostat with Azacitidine in Patients with Myelodysplastic Syndrome

This open-label, phase 1/2 trial will assess the safety of mocetinostat in combination with azacitidine for the treatment of patients with intermediate- or high-risk myelodysplastic syndrome (MDS). Patients aged ≥18 years with an Eastern Cooperative Oncology Group performance status 0 or 1; a diagnosis of intermediate- or high-risk MDS per the International Prognostic Scoring System; and who do not have a history of small, moderate, or large pericardial effusion, tamponade, and/or pericarditis may be eligible to enroll if other criteria are met. Eligible patients will receive combination therapy with mocetinostat plus azacitidine.

The primary outcome measures are the number of patients with adverse events, including pericardial events, as a measure of safety. The secondary outcome measures are the number of patients experiencing clinical disease responses. This study expects to enroll 20 patients at multiple locations across the United States. For more information, contact Mirati Therapeutics Study Locator Services at 844-356-0895 or miratistudylocator@emergingmed.com. The NLM Identifier is NCT02018926.

8 Allo versus Hypomethylating/Best Supportive Care for Myelodysplastic Syndrome

Investigators in this multicenter trial seek to compare reduced-intensity allogeneic hematopoietic stem-cell transplantation (HSCT) with hypomethylating therapy versus reduced-intensity allogeneic HSCT with best supportive care in patients with intermediate-2 and high-risk myelodysplastic syndrome (MDS). Patients aged 50 to 75 years with de novo MDS who have, or have previously had, intermediate-2 or high-risk disease per the International Prognostic Scoring System, and a Karnofsky performance status >70 or Eastern Cooperative Oncology Group performance status ≤1 may be eligible for enrollment if other criteria are met. Eligible patients will receive allogeneic HSCT with either hypomethylating therapy or with best supportive care.

The primary outcome measures are overall survival probabilities over 3 years. Secondary outcome measures are leukemia-free survival and patient quality of life. This study expects to enroll 400 patients at multiple locations across the United States. For more information, contact Heather Wittsack at 301-251-1161 or hwittsack@emmes.com, or Adam Mendizabal, PhD, at 301-251-1161 or amendizabal@emmes.com. The NLM Identifier is NCT02016781.

9 Chemotherapy for Myelodysplastic Syndrome Before Stem-Cell Transplantation

The purpose of this randomized, open-label, phase 2 clinical trial is to study different chemotherapies for treating patients with myelodysplastic syndrome (MDS) before receiving donor stem-cell transplantation. Patients aged ≥18 years with a diagnosis of de novo or secondary MDS—including chronic myelomonocytic leukemia—as defined by the 2008 World Health Organization classification system, who have not had previous treatment for MDS with an intensive chemotherapy regimen or with a hypomethylating agent, and who are considered candidates for transplants may be eligible to enroll if other criteria are met. Eligible patients will be randomized to treatment arm A and receive cytarabine, idarubicin, or daunorubicin hydrochloride, or treatment arm B and receive decitabine or azacitidine.

The primary outcome measures are survival free of failure (ie, death or disease relapse) over 18 months. Secondary outcome measures include changes in quality-of-life scores per the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire–Core 30, frequency at which the patients undergo transplantation, and overall survival. This study expects to enroll 60 patients in Seattle, WA. For more information, contact Bart L. Scott at 206-667-1990, or Eric Y. Chen at 206-326-3111. The NLM Identifier is NCT01812252.

10 Phase II Decitabine versus Azacitidine for Myelodysplastic Syndrome

Investigators in this phase 2, randomized, open-label study will compare how decitabine and azacitidine help to control myelodysplastic syndrome (MDS) when given on a shorter-than-standard dosing schedule. The safety of these drugs will also be examined. Patients aged ≥18 years with de novo or with secondary International Prognostic Scoring System low- or intermediate-1–risk MDS, and an Eastern Cooperative Oncology Group performance status ≤3 may be eligible for enrollment if other criteria are met. Eligible patients will be randomized to receive either best supportive care, azacitidine 75 mg/m2 intravenously (IV) or subcutaneously daily for 3 or 5 days every 28 days, or decitabine 20 mg/m2 IV daily for 3 days every 28 days.

The primary outcome measures are event-free survival, defined as the time from the beginning of treatment until the occurrence of an event or until the last follow-up. Secondary outcome measures are the overall improvement rate, defined as complete remission, partial remission, marrow complete remission, or hematologic improvement, measured using each patient’s best response to the 2 different agents. This study expects to enroll 240 patients at multiple locations across the United States. For more information, contact Elias Jabbour, MD, at 713-792-4764. The NLM Identifier is NCT02269280.

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