Clinical Trials Report for April 2014 | Aplastic Anemia & MDS International Foundation Return to top.

Clinical Trials Report for April 2014

We have highlighted some clinical trials that were opened since the beginning of 2014. There are many others that are also underway. You can find them on ClinicalTrials.gov. Each trial has a specific purpose related to aplastic anemia, MDS, or PNH. Should you consider a clinical trial? Ask your doctor or contact the study coordinators for further information. Learn more in the Clinical Trials section.

Search for aplastic anemia studies actively recruiting patients. The following aplastic anemia studies are a sample of the 75 studies that are actively recruiting aplastic anemia patients.

Title/Link to more information

Study Purpose

Study Coordinator

Eltrombopag for Moderate Aplastic Anemia

To evaluate the safety and effectiveness of eltrombopag in people with moderate aplastic anemia who need treatment for significantly low blood cell counts.

National Institutes of Health Clinical Center

Contact: Ronan Desmond, M.D.    301-451-7143  

desmondrg@mail.nih.gov

Please refer to this study by its ClinicalTrials.gov identifier: NCT01328587
A Description of Bacteria in the Mouths of Patients With Severe Aplastic Anemia This research is being done to describe the types of bacteria found in the mouths of patients who have severe aplastic anemia (SAA) and are treated with drugs that suppress the immune system or with stem cell transplant. People with SAA who receive these treatments are more likely to get infections. Studies show that there might be a link between the bacteria in your mouth and those bacteria that can cause infections. The bacteria found in the mouths of patients with SAA will be described.

National Institutes of Health Clinical Center

Contact: Nancy Ames, R.N. (301) 451-0565 names@mail.cc.nih.gov

Please refer to this study by its ClinicalTrials.gov identifier: NCT01900119
Post Transplant Cyclophosphamide Cytoxan) for GvHD Prophylaxis The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD). The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS) following allo HCT and assess the safety of post-transplant cyclophosphamide (cytoxan) for MUD and MMUD transplantation. Disease recurrence and time to recurrence in patients receiving post-transplant cyclophosphamide compared to historical control without post-transplant cyclophosphamide (cytoxan) will also be evaluated. Other objectives will be to determine the time of onset, severity, responsiveness to treatment, organs involved of acute and chronic GVHD as well as observation of Immune Reconstitution over time.

UAB Bone Marrow Transplantation and Cellular Therapy Program

Contact: Lisa D Williams, RN  205-934-0066
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells Primary endpoint of this study is chronic GVHD by one year. Secondary outcomes measures include transplant related mortality, engraftment, degree of donor-host chimerism, incidence of acute and chronic graft versus host disease (GVHD), transplant related morbidity and overall survival.

National Institutes of Health Clinical Center, Bethesda, Maryland

Contact: Elena J Cho (301) 594-8013 elena.cho@nih.gov

Please refer to this study by its ClinicalTrials.gov identifier: NCT01174108

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 300 studies that are actively recruiting MDS patients.

Title/Link to more information

Study Purpose

Study Coordinator

Clofarabine or Daunorubicin Hydrochloride and Cytarabine Followed By Decitabine Observation in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia

This randomized phase III trial studies clofarabine to see how well it works compared with daunorubicin hydrochloride and cytarabine when followed by decitabine or observation in treating older patients with newly diagnosed acute myeloid leukemia. Drugs used in chemotherapy, such as clofarabine, daunorubicin hydrochloride, cytarabine, and decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more cancer cells. It is not yet known which chemotherapy regimen is more effective in treating acute myeloid leukemia.

This study is being conducted in 273 sites across the U.S. Find the contact information for the study nearest you.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02085408

The Efficacy and Safety of Oral Azacitidine Plus Best Supportive Care Versus Placebo and Best Supportive Care in Subjects With Red Blood Cell (RBC) Transfusion-Dependent Anemia and Thrombocytopenia Due to International Prognostic Scoring System (IPSS) Low Risk Myelodysplastic Syndrome (MDS) A Phase 3 evaluation of the Efficacy and Safety of oral Azacitidine plus best supportive care versus placebo and best supportive care in subjects with red blood cell (RBC) transfusion-dependent anemia and thrombocytopenia due to International Prognostic Scoring System (IPSS) lower risk myelodysplastic syndromes (MDS).

Multiple Locations

Contact: Associate Director, Clinical Trial Disclosure 1-888-260-1599

ClinicalTrialDisclosure@celgene.com               

Please refer to this study by its ClinicalTrials.gov identifier: NCT01566695
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant This study is a research study involving subjects who are diagnosed with a malignant disease, that has either failed standard therapy or is unlikely to be cured with standard non-transplant therapy, who will receive a peripheral blood stem cell transplant. A malignant disease includes the following: Chronic Myeloid Leukemia (CML) in chronic phase, accelerated phase or blast crisis; Acute Myelogenous Leukemia (AML); Myelodysplastic Syndrome (MDS); Juvenile Myelomonocytic Leukemia (JMML); Acute Lymphoblastic Leukemia (ALL); or Lymphoma (Hodgkin's and Non-Hodgkin's) The purpose of this study is to learn more about the effects of CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents and young adults. CD34+ stem cells are the cells that make all the types of blood cells in the body. GVHD is a condition that results from a reaction of transplanted donor T-lymphocytes (a kind of white blood cell) against the recipient's body and organs. Study subjects will be offered treatment involving the use of a Miltenyi CliniMacs CD34+ selection device to remove T-cells from a peripheral blood stem cell transplant in order to decrease the risk of acute and chronic GVHD.

Contact: Jean Sosna, RN   
212-305-2050 

Please refer to this study by its ClinicalTrials.gov identifier: NCT02061800

Myelodysplastic Syndromes (MDS) Event Free Survival With Iron Chelation Therapy Study (TELESTO) The primary purpose of this study is to prospectively assess the efficacy and safety of iron chelation therapy with deferasirox compared to placebo in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload.

This study is being conducted in 187 sites across the U.S. Please click here to find the contact information for the study nearest you.

Contact: Novartis Pharmaceuticals 1-888-669-6682

Please refer to this study by its ClinicalTrials.gov identifier: NCT00940602

Search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. No PNH studies opened between January 1, 2014 and February 24, 2014.

Title/Link to more information

Study Purpose

Study Coordinator

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH).

Johns Hopkins University Medical Center , Baltimore

Contact: Lynn Sanders 203-439-9609 sandersl@alxn.com

Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360
Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This Phase 1 study is to assess the safety and tolerability of a single dose of TT30.

Safety and tolerability will be evaluated in all subjects by physical exam, vital signs, ECGs, laboratory changes over time, adverse events, and antibody development.

This study is being conducted in multiple sites across around the world. Find the contact information for the study nearest you.

In the United States, the study is at the USC/Norris Comprehensive Cancer Center

Contact: Laurie Hornor, RN   323-864-3978

Laurie.Hornor@med.usc.edu  

Please refer to this study by its ClinicalTrials.gov identifier: NCT01566695
Mismatched Family Member Donor Transplantation for Children and Young Adults With High Risk Hematological Malignancies The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in this high risk patient population by 1) limiting the complication of graft versus host disease (GVHD), 2) enhancing post-transplant immune reconstitution, and 3) reducing non-relapse mortality.

St. Jude Children's Research Hospital, Memphis, TN

Contact: Brandon Triplett, MD 866-278-5833   info@stjude.org

Please refer to this study by its ClinicalTrials.gov identifier: NCT00566696
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