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

Clinical Trials Report for December 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 62 open studies recruiting aplastic anemia patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Extended Dosing with Eltrombopag for Severe Aplastic Anemia The main goal of this study is to determine if whether 6 months of treatment with eltrombopag for Severe Aplastic Anemia This study is being conducted at the National Institutes of Health Clinical Center

Contact: Marlene Peters-Lawrence, R.N
(301)-443-6144  mpeters@nhlbi.nih.gov

Contact: Thomas Winker, MD
(301)-451-7142  winklert@nhlbi.nih.gov

Please refer to this study by its ClinicalTrials.gov identifier: NCT01891994
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies. Giving low doses of chemotherapy, such as cyclophosphamide and fludarabine phosphate, before a donor stem cell transplant may help stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Reducing the intensity of the chemotherapy and radiation may also reduce the side effects of the donor stem cell transplant. This study is being conducted at Thomas Jefferson University

Contact: Dolores Grosso, DNP, CRNP, 215-955-8874       

Contact: Donna Zuccarello, 215-503-0226       

Please refer to this study by its ClinicalTrials.gov identifier: NCT02566304
Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia The study has a Fludarabine-based preparative regimen followed by an allogeneic hematopoietic stem cell transplant using related or unrelated donor in persons 0-70 years of age diagnosed with dyskeratosis congenita or severe aplastic anemia who have bone marrow failure characterized by a requirement for red blood cell and platelet transfusions. Three different preparative regimens are included based on disease and donor type. This study is being conducted at the University of Minnesota.

Contact: Timothy Krepski 612-273-2800
Tkrepsk1@fairview.org

Please refer to this study by its ClinicalTrials.gov identifier: NCT02162420

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 309 studies recruiting MDS patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS This study is targeted for immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS). This study is being conducted by New York Medical College and the Medical College of Wisconsin/Children’s Hospital of Wisconsin

New York Medical College   

Valhalla, New York
Contact: Sandra Fabricatore, RN, PNP,    914-594-2152    sandra.fabricatore@wmchealth.org  
Contact: Erin Morris, RN, 714-964-5359    erin_morris@nymc.edu 

Medical College of Wisconsin/Children's Hospital of Wisconsin   

Milwaukee, Wisconsin
Contact: Julie A Talano, MD, 414-955-4185    jtalano@mcw.edu  
Contact: Kathy Jodarski, 414-266-2681    kjodarski@chw.org  

Please refer to this study by its ClinicalTrials.gov identifier: NCT02221310
IL-15 Super Agonist ALT-803 to Treat Relapse Of Hematologic Malignancy After Allogeneic SCT

This is a multi-center, phase I/II clinical trial for patients who have relapsed more than 60 day after allogeneic transplant for a hematologic malignancy. The study consists of two phases. The dose finding phase is a modified version of a phase I trial and the extended phase is a modified version of a phase II trial. The primary objective of the dose finding phase is to determine the maximum tolerated, minimum efficacious dose (MTD/MED) of a interleukin-15 (IL-15) super agonist complex (ALT-803) when given once weekly for 4 weeks in the outpatient setting. The study will follow a standard 3+3 design of dose escalation for toxicity with an added feature of stopping early if efficacy is confirmed. There are six dose levels of ALT-803 for to determine the MTD/MED: 1, 3, 6, 10, 20, and 30 mcg/kg.

This study is being conducted is currently being conducted at the University of Minnesota.
Masonic Cancer Center,
University of Minnesota   
Minneapolis, Minnesota
Contact: Judith Witte, RN, 612-626-0169    mirab001@umn.edu  
   
Please refer to this study by its ClinicalTrials.gov identifier: NCT01885897
Interventional Validation of an MDS-Specific Measure of Quality of Life: Assessing the Responsiveness of the Quality of Life in Myelodysplasia Scale (QUALMS-1) to Different Hypomethylating Agent Regimens for Low and Intermediate Risk Disease


The goal of this research study is to test a quality-of-life questionnaire called QUALMS-1 in patients with MDS. This study is being conducted in FL, MD,
MA, NY,OH, and TX.

Contact: Guillermo Garcia-Manero, MD
713-745-3428
   
Please refer to this study by its ClinicalTrials.gov identifier: NCT02378701

 

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