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 clinical trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a… 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 79 open studies recruiting aplastic anemia patients in the United States.
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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 cyclophosphamide: Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system. with partially HLA HLA: See human leukocyte antigen. -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 311 studies recruiting MDS patients in the United States.
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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 Acute Myeloid Leukemia: (uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood cells and platelets. Some cases of MDS become… | 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 chemotherapy: (kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with… . | 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 |
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Myelodysplastic Syndromes (MDS) Event Free Survival With
Iron Chelation Therapy
Iron Chelation Therapy:
(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S. - deferasirox, an oral iron chelator,…
Study |
The primary purpose of this study is to prospectively assess the efficacy and safety of iron chelation therapy with deferasirox deferasirox: It comes in a tablet form which is dissolved in juice or water and taken by mouth. Iron build-up can occur in patients who get a lot of red blood cell transfusions. Deferasirox binds with the iron in the body and is removed by the kidneys. Bone marrow failure disease patients are at risk for… compared to placebo placebo: A placebo is an inactive pill, liquid, or powder that has no treatment value. in patients with myelodysplastic syndromes (low/int-1 risk) and transfusional iron overload iron overload: A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated. . | This study is being conducted by Novartis Pharmceuticals Contact: 1-888-669-6682 Please refer to this study by its ClinicalTrials.gov identifier: NCT00940602 |
| 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 7 studies recruiting PNH patients in the United States.
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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 |