The following clinical trials are currently recruiting participants with myelodysplastic syndrome (MDS) for inclusion. Each trial description includes the NLM Identifier to use as reference with Clinical Trials.gov.
1. Alemtuzumab in Patients with MDS
The goal of this phase 2, open-label clinical trial is to assess the safety and efficacy of alemtuzumab in patients with MDS. Alemtuzumab is a humanized immunoglobulin G1 monoclonal antibody that destroys specific types of lymphocytes by targeting the CD52 protein.
Participants will receive a test dose of 1 mg of alemtuzumab intravenously. If the test dose is tolerated, then patients will receive 10-mg doses intravenously for a 10-day treatment cycle. Patients will receive blood transfusions as needed. After completion of the trial, patients are required to return for follow-up at 1 month, 3 months, 6 months, and annually for 5 years. To be eligible for participation, individuals must be aged 18 to 72 years, have a diagnosis of MDS, have anemia requiring blood transfusion, and should not be receiving other therapies for MDS (except filgrastim, erythropoietin, or other transfusion support) for at least 4 weeks.
The primary outcome of this trial is complete or partial hematologic response measured at 3 months after the first dose of alemtuzumab and sustained on ?2 serial measurements performed 1 month apart. Other outcome measures include transfusion independence for red blood cells and/or platelets, overall survival, life-threatening toxicity, and transformation-free survival. This trial is expected to enroll 78 patients and will be conducted at the National Institutes of Health Clinical Center in Bethesda, MD. For more information, contact Barbara Weinstein, RN, at 301-594-4180 or
2. Pracinostat with Azacitidine for Previously Untreated MDS
The purpose of this phase 2, randomized, double-blind trial is to determine the safety and efficacy of pracinostat plus azacitidine compared with placebo plus azacitidine in patients with previously untreated MDS. Patients in the experimental treatment arm will receive 60 mg of pracinostat orally 3 times a week for 3 weeks followed by 1 week of rest. Patients in the comparator arm will receive placebo orally 3 times a week for 3 weeks followed by 1 week of rest. Patients in both arms will be also administered azacitidine 75 mg/m2 subcutaneously or intravenously for 7 days of each 28-day cycle; treatment cycles will repeat. The trial is expected to enroll 100 patients.
The primary outcome is efficacy at 6 months measured by complete remission rate. Other outcome measures include overall response rate and overall survival, hematologic improvement, duration of response, progression-free survival, rate of leukemic transformation, and adverse event profile. Patients aged ?18 years with histologic evidence of MDS, bone marrow aspiration and biopsies within 28 days of the first study treatment, no previous treatment with hypomethylating agents, and clinical indication for azacitidine are eligible to participate in the trial if other criteria are met. This trial will be conducted at sites throughout the United States. For more information, please contact Guillermo Garcia-Manero, MD, at 713-792-2121, MD Anderson Cancer Center in Houston, TX. The NLM Identifier is NCT01873703.
3. Safety and Efficacy of INCB024360 in Patients with MDS
In this phase 2, open-label, single-group trial, investigators are evaluating the safety and efficacy of INCB024360 in patients with MDS, and the long-term outcomes of MDS after therapy with INCB024360, an inhibitor of the enzyme indoleamine 2,3-dioxygenase, is discontinued. The trial is expected to recruit 40 patients, and participants will receive the study drug in 28-day cycles. The duration of the study comprises the treatment phase followed by a 24-month follow-up period. To be eligible to participate in this trial, patients must be aged ?18 years with a confirmed diagnosis of MDS and should meet other inclusion and exclusion criteria.
Trial investigators will assess the overall response rate from time on treatment through follow-up (approximately 36 months). Secondary outcome measures include time to acute myeloid leukemia progression, overall survival, and number of patients with serious adverse events. This trial will be conducted at the H. Lee Moffitt Cancer Center and Research Institute in Tampa, FL. For more information, contact Lisa Nardelli at 813-745-4731 or
4. Decitabine and Vorinostat Conditioning for High-Risk MDS
The purpose of this phase 2, open-label, single-group trial is to facilitate natural killer (NK)-cell survival and expansion by administering decitabine and vorinostat to patients with high-risk MDS. After receiving 10 mg/m2 of intravenous decitabine daily on days 1 to 5, and 200 mg of vorinostat orally twice daily on days 6 to 15, patients will receive a single infusion of CD3-enriched and CD19-enriched donor NK cells intravenously on day 17, and a short infusion of interleukin-2 administered subcutaneously 3 times a week for 3 doses beginning on day 17. A second cycle of treatment will be repeated 6 to 8 weeks following the cycle 1 start date.
Primary outcome measure is the objective response rate after 2 courses of treatment. Secondary outcome measures include safety and tolerability, number of patients who become transfusion independent, NK-cell expansion, and overall survival. Patients aged 18 to 75 years with high-risk MDS, who have had ?2 cycles of hypomethylating agents (ie, azacitidine or decitabine) without evidence of treatment failure are eligible to participate in this study if other criteria are met. This clinical trial is expected to enroll 46 patients and will be conducted at the University of Minnesota Masonic Cancer Institute in Minneapolis. For more information, contact Erica Warlick, MD, at 612-625-5467 or
5. Bioequivalence of Azacitidine in Patients with MDS
The goal of this phase 1, randomized, open-label trial is to determine the bioavailability of azacitidine injection compared with the bioavailability of Vidaza (azacitidine) in patients with MDS. This clinical trial uses a crossover model and is expected to enroll 36 participants. Patients in the first treatment arm will receive azacitidine followed by Vidaza, both dosed subcutaneously at 75 mg/m2 on day 1 of either cycle 1 or cycle 2 per randomization assignment. Patients in the second treatment arm will receive Vidaza followed by azacitidine, both dosed subcutaneously at 75 mg/m2 on day 1 of either cycle 1 or cycle 2 per randomization assignment. To be eligible to participate in this study, patients must be aged ?18 years, diagnosed with MDS, and prescribed Vidaza, and should meet additional criteria.
The primary outcome of this trial is the measurement of azacitidine in plasma samples to determine Cmax, AUC0-t, and AUC0-? at 13 time points from predose to 8 hours after dosing. The secondary outcomes are safety and tolerability, measured by the number of patients with adverse events. Trial locations in the United States include California and Florida. For more information regarding recruitment in California, contact Veena Charu, MD, at 714-999-1465 or
6. Eltrombopag in Patients with Low-to-Intermediate Risk MDS
The purpose of this phase 2, open-label, single-group trial is to determine whether eltrombopag can improve platelet counts and whether it is safe in patients with MDS. Eltrombopag mimics the protein thrombopoietin, which stimulates platelet production, and has been associated with increasing platelet counts in patients with chronic idiopathic thrombocytopenic purpura. The trial is expected to enroll 30 patients. Participants will receive eltrombopag orally once daily for 90 days; they will be monitored weekly for platelet counts and every 4 weeks for other clinical evaluations. If platelet counts improve after 90 days, additional doses of eltrombopag will be added to the treatment regimen.
Primary outcomes are an increase in the platelet count of 20,000/µL from baseline as well as toxicity profile at 3 months. Secondary outcomes include any changes in the platelet count, changes in platelet transfusion requirements, incidence of bleeding, and changes in serum thrombopoietin level. Patients aged ?18 years with a confirmed diagnosis of MDS and platelet count of ?30,000/µL are eligible to participate in the trial if other criteria are met. This trial is located at the National Institutes of Health Clinical Center in Bethesda, MD. For more information, contact Barbara Weinstein, RN, at 301-594-4180 or
7. Efficacy and Safety of Oral Rigosertib in MDS
In this phase 2, open-label, single-group trial, investigators are evaluating the safety of rigosertib and whether it is associated with a reduction in the number of blood transfusion units needed in patients with MDS that is classified as low, intermediate-1, or trisomy 8 intermediate-2, and who are transfusion-dependent. It is expected that 60 patients will be enrolled in this study. Participants will receive 560 mg of rigosertib orally twice daily over a 14-day treatment course. The primary outcome of this trial is the number of red blood cell transfusion units during an 8-week time frame. Other outcome measures include adverse events, change in the number of bone marrow blasts, and complete blood counts.
Patients aged ?18 years with a confirmed diagnosis of MDS and transfusion dependency defined as ?4 units of red blood cells within 8 weeks before baseline are eligible to participate if other criteria are met. Trial locations include Arizona, Georgia, Minnesota, New York, and South Carolina. For more information, contact François E. Wilhelm, MD, PhD, at 609-281-7086 or
8. Treosulfan and Fludarabine Phosphate with or without Total Body Irradiation in Patients with MDS
The goal of this phase 2, randomized, open-label trial is to assess the efficacy of treosulfan and fludarabine phosphate with or without total body irradiation (TBI) prior to donor stem-cell transplant in patients with MDS. Patients in the first experimental treatment arm will receive treosulfan intravenously for 2 hours at 4 to 6 days before transplant (day 0) and fludarabine phosphate intravenously for 30 minutes at 2 to 6 days before transplant (day 0). Patients in the second experimental treatment arm will receive treosulfan and fludarabine phosphate as in the first treatment arm, and will also undergo low-dose TBI on day 0. Patients in both treatment arms will undergo allogeneic stem-cell transplant or bone-marrow transplant on day 0.
The primary outcome is progression-free survival at 6 months posttransplant. Other outcome measures include changes in gene- expression profiles, relapse risk, incidence of relapse or progression, nonrelapse mortality, overall survival, and incidence of acute or chronic graft-versus-host disease. This trial is expected to enroll 80 patients. To qualify for participation, patients must have donors who meet specified requirements, must be aged ?18 years with a diagnosis of MDS, and should meet other inclusion and exclusion criteria. This clinical trial will be conducted at the Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium in Seattle, WA. For more information about this study, contact Principal Investigator H. Joachim Deeg at 206-667-5985. The NLM Identifier is NCT01894477.
9. Event-Free Survival with Iron Chelation Therapy in Patients with MDS
The purpose of this phase 2, randomized, double-blind trial is to prospectively evaluate the efficacy and safety of iron chelation therapy with deferasirox compared with placebo in patients with MDS and transfusional iron overload. This trial is expected to recruit 210 participants. Patients in the experimental arm will receive deferasirox, which will be provided as 125-mg, 250-mg, and 500-mg tablets. Patients in the comparator arm will receive placebo tablets. Patients aged ?18 years with low-risk to intermediate-1–risk MDS, weighing between 35 kg and 135 kg, and with a ferritin level of >1000 µg/L are eligible to participate if other criteria are met.
The primary outcome is event-free survival assessed up to 5 years after study randomization. Several secondary outcome measures include hematologic improvement, overall survival, proportion of patients with hypothyroidism, and disease progression. This trial will be conducted throughout the United States. For more information, contact Novartis Pharmaceuticals at 888-669-6682. The NLM Identifier is NCT00940602.
10. Investigational Drug PF-04449913 in Patients with Refractory or Relapsed MDS
In this phase 2, open-label, single-arm trial, investigators are assessing the safety and efficacy of a hedgehog inhibitor, PF-04449913, in patients with refractory or relapsed MDS. Patients in the experimental arm will receive 100 mg of PF-04449913 orally once daily in 4-week cycles for a total of 4 cycles, followed by an optional continuation phase. This study will be divided into 2 stages: 20 patients will be enrolled in the first stage, and if ?2 patients respond in this stage, then 15 additional patients will be enrolled in the second stage.
The primary outcome is the overall response rate during a 6-month time period. Other outcome measures include number of patients with overall survival, event-free survival, and serious adverse events, and the median time to transformation to acute myeloid leukemia. To qualify for trial participation, patients must be aged ?18 years with a confirmed diagnosis of MDS that is refractory to previous therapy with hypomethylating agents (ie, azacitidine and/or decitabine); they must also meet other criteria. This trial is expected to enroll 35 patients and will be conducted at the H. Lee Moffitt Cancer Center and Research Institute in Tampa, FL. For more information, contact Lisa Nardelli at 813-745-4731 or