Clinical Trials Report for January 2016 | Aplastic Anemia and MDS International Foundation

Clinical Trials Report for January 2016

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 78 open studies recruiting aplastic anemia patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome The purpose of this study is to assess efficacy and safety of BL-8040 on top of standard immunotherapy regimen of hATG, cyclosporine and steroids in patients with Hypoplastic MDS and AA over the course of a six month (180 day) treatment period. This study is being conducted at
MD Anderson Cancer Center

Contact: Lisa Stepner, RN   
972-25489100 ext 159   
lisas@biolinerx.com
   
Contact: Dana Keinan, PhD   
972-25489100 ext 147
danak@biolinerx.com  

Please refer to this study by its ClinicalTrials.gov identifier: NCT02462252
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
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders 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 Fred Hutchinson Cancer Research Center/ University of Washington Cancer Consortium in Seattle, WA and the Children’s Hospital of Wisconsin in Milwaukee, WI.

Fred Hutchinson Cancer Research Center
Contact: Lauri Burroughs, MD
 206-667-2396
lburroug@fredhutch.org  


Children’s Hospital of Wisconsin
Contact: Julie-An M. Talano, MD
414-955-4170
jtalano@mcw.edu


Please refer to this study by its ClinicalTrials.gov identifier: NCT00919503

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 311 studies recruiting MDS patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Study to Evaluate Imetelstat (JNJ-63935937) in Subjects With International Prognostic Scoring System (IPSS) Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) The purpose of this study is to evaluate the efficacy and safety of imetelstat in transfusion dependent participants with low or intermediate-1 risk myelodysplastic syndrome (MDS) that is relapsed/refractory to erythropoiesis-stimulating agent (ESA) treatment. This study is being conducted in multiple locations both nationally and internationally and is now recruiting in selected locations.

Contact: Janssen Research and Development, LLC
JNJ.CT@sylogent.com


Please refer to this study by its ClinicalTrials.gov identifier: NCT02598661
Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral CDAi in Patients With MDS)

This 2-stage, open-label study will evaluate safety and pharmacokinetics of ASTX727, as well as determine the dose for the study's second stage. In the second stage the selected dose will be confirmed and evaluated for clinical activity, including response rate.

This study is being conducted is currently being conducted in AZ, CA, IL, IN, MD, MA, NJ, NY, TN, TX, WI, and in different regions in Canada

Contact: Roya Nawabi
Roya.nawabi@astx.com

Contact: Jacqueline Ames
Jacqueline.ames@astx.com

Please refer to this study by its ClinicalTrials.gov identifier: NCT02103478
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