Clinical Trials Report for November 2014 | Aplastic Anemia and MDS International Foundation

Clinical Trials Report for November 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.

To find out more about clinical trials at specific institutions near you, check their websites. Large academic healthcare institutions often identify the clinical trials being run by their researchers. Here is an example from MD Anderson.

Search for aplastic anemia studies actively recruiting patients. The following are a sample of the 67 studies that are actively recruiting aplastic anemia patients.

Title/Link to more information

Study Purpose

Study Coordinator

Fertility Preservation in Women Who Will Have Gonadotoxic Therapy or Hematopoetic Stem Cell Transplantation, and in Women With Sickle Cell Disease Some treatments for cancer or other diseases can lead to infertility in women. These treatments include chemotherapy, some stem cell transplants, and pelvic radiotherapy. They are called gonadotoxic therapies. Women can now have their eggs frozen before they have these treatments. This may allow them to get pregnant later. Researchers want to learn more about this technology and processes.
This study if for women at least 18 years old who are past puberty and before menopause. They must be scheduled to have gonadotoxic therapies.
National Institutes of Health Clinical Center

Contact: Christine L Atallah, R.N.   
(301) 435-7926 christine.atallah@nih.gov
   
Please refer to this study by its ClinicalTrials.gov identifier: NCT02225145

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 285 open studies that are actively recruiting MDS patients.

Title/Link to more information

Study Purpose

Study Coordinator

Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) in Patients With Myelodysplastic Syndromes

The purpose of this study is to see if selinexor will improve the blood counts and bone marrow function in people with your type of MDS. This study is being conducted at Memorial Sloan Kettering Cancer Center in NY.
Contact: Virginia Klimek, MD, 212-639-6519 
Please refer to this study by its ClinicalTrials.gov identifier: NCT02228525



A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS) The purpose of the study is to test the safety of six cycles of cenersen treatment and to begin to test the hypothesis that intermittent administration of cenersen may lead to a reduced dependence on transfusion. This study is being conducted at the H. Lee Moffitt Cancer Center
& Research Institute, Tampa FL
 Contact: Jennifer Cooksey, RN Jennifer.Cooksey@moffitt.org 
     
Please refer to this study by its ClinicalTrials.gov identifier: NCT02243124
Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)

The purpose of this trial is to examine the hematologic response rate of Exjade® in patients with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed iron chelator, deferasirox has the advantage of oral administration. This study is being conducted at the Abramson Cancer Center of the University of Pennsylvania, Philadelphia PA
Contact: James Mangan, MD, PhD, 855-216-0098, PennCancerTrials@emergingmed.com
Please refer to this study by its ClinicalTrials.gov identifier: NCT02233504
An Open Label Phase II Pharmacokinetic and Pharmacodynamic Assessment of the Potential for QTc Prolongation Following First Induction Treatment With CPX-351 (Cytarabine:Daunorubicin) Liposome Injection in Acute Leukemias and MDS Patients The purpose of this study is to assess the effects of CPX-351 on cardiac repolarization following the first induction cycle of CPX-351, assess total drug levels, assess total and encapsulated drug levels (in a subset of patients), and asses copper levels in patients with normal to mild hepatic and renal impairment. A correlation will be made between changes in cardiac repolarization and drug levels of CPX-351. This study is being conducted at Franciscan Saint Francis
Health, Indianapolis, IA
Contact: Arthur C Louie, MD, 609.243.0123, alouie@celatorpharma.com
Please refer to this study by its ClinicalTrials.gov identifier: NCT02238925

Search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. The following are a sample of the 11 open studies for PNH patients in the United States.

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