Clinical Trials Report for August 2015 | Aplastic Anemia and MDS International Foundation

Clinical Trials Report for August 2015

We have highlighted some clinical trials currently recruiting patients. Each has a specific purpose related to aplastic anemia, MDS, or PNH. These clinical trials (also known as research studies) were obtained from www.clinicaltrials.gov, the federal web site that presents information on clinical trials worldwide. To narrow down your search criteria, use the advanced search feature found on the home page.

  • If you want to see only studies that are currently recruiting patients, select that option from the recruitment field at the beginning of the advanced search.
  • To locate a clinical trial in your state or region, simply select this from the locations field in the middle of the page.
  • To search in predetermined time period, indicate the starting and ending dates in the date received field at the bottom of the page.
  • There are many more criteria available to search in www.clincial trials.gov through its advanced search features.

Should you consider a clinical trial?  Ask your doctor or contact the trial’s study coordinators for further information. Learn more in the Clinical Trials section.

Search for aplastic anemia studies actively recruiting patients. The following are a sample of the 74 open studies recruiting aplastic anemia patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Transplantation of Partially Mismatched Related or Matched Unrelated Bone Marrow for Patients With Refractory Severe Aplastic Anemia

The main goal of this study is to determine if it is feasible for SAA patients to be transplanted using non-myeloablative conditioning and post transplantation cyclophosphamide with partially HLA-mismatched donors. This study is being conducted at Sidney Kimmel Comprehensive Cancer Center in Baltimore, Maryland.

Contact: Amy DeZern, MD
410-502-7208  adezern1@jhmi.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT02224872
Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia

The purpose of this study is to analyze the effect of eltrombopag given to patients with moderate to very severe aplastic anemia. This study is being conducted at the University of Utah in Salt Lake City, Utah

Contact: Renee Rinaldi, BS 801-585-5341 renee.rinaldi@hsc.utah.edu or Marc Nuttall, MD, marc.nuttall@hsc.utah.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT01703169

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 288 studies recruiting MDS patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Ipilimumab in Treating Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia The primary purpose of this study is to evaluate the safety and toxicity associated with the administration of ipilimumab in terms of dose limiting toxicities (DLT), and maximally-tolerated dose (MTD) in a cohort of patients with high risk myelodysplastic syndrome who failed hypomethylating therapy, and patients with acute myeloid leukemia (AML) who underwent induction therapy but are not planned for further intensive chemotherapy. This study is being conducted by the National Cancer Institute in Connecticut, Maryland, Missouri, New York, North Carolina, Texas

Yale New Haven Hospital North Haven Medical Center—contact Steven D. Gore, MD 203-737-7103 steven.gore@yale.edu  

Johns Hopkins University/Sidney Kimmel Cancer Center—contact Doug Smith, MD, 410-614-5068 bdsmith@jhmi.edu

Washington University—contact Mark A. Schroeder, MD 314-454-8304 mschroed@dom.wustl.edu
  
Columbia University Medical Center—contact Mark G. Frattini 212-851-4872 mgf2122@columbia.edu  

University of North Carolina at Chapel Hill—contact Joshua Zeidner 919-962-5164 Joshua_Zeidner@med.unc.edu  

Duke University Medical Center—contact Herbert Hurwitz 919-681-6006 Herbert.Hurwitz@dm.duke.edu  

Texas Oncology at Baylor Irving Cancer Center—contact Moshe Levy 214-370-1033 mylevy@gmail.com  

Please refer to this study by its ClinicalTrials.gov identifier: NCT01757639
Combination Study of Deferasirox and Erythropoietin in Patients With Low- and Int-1-risk Myelodysplastic Syndrome.


The primary purpose of this trial is to assess the effect of treatment with deferasirox combined with erythropoietin vs. erythropoietin alone on erythropoiesis in patients with low- and int-1-risk myelodysplastic syndrome. This study is being conducted by Novartis Pharmceuticals

Contact: 1-888-669-6682

Please refer to this study by its ClinicalTrials.gov identifier: NCT01868477

A Study To Evaluate PF-04449913 With Chemotherapy In Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome


This is a study to evaluate PF-04449913 (an inhibitor of the Hedgehog pathway) in Acute Myeloid Leukemia and high-risk Myelodysplastic Syndrome in combination with standard agents used to treat these diseases This study is being conducted in AL,
CA, FL, GA, IL, KS, MD, MO, NJ, NY, OH,
TN, TX, WA, and Canada.

Contact: Pfizer CT. gov Call Center
Esperanza Papadopoulos, M.D
1-800-718-1021   
Please refer to this study by its ClinicalTrials.gov identifier: NCT01546038

 

Please search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. The following are a recent sample of the 8 studies recruiting PNH patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH This study will be the initial exploration of APL-2 in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of APL-2 will guide decisions to further develop the drug. This study is being conducted at the University of Louisville, KY

Contact: Candace Depp        pnh@apellis.com

Please refer to this study by its ClinicalTrials.gov identifier: NCT02264639