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.clincialtrials.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 76 open studies recruiting aplastic anemia patients in the United States.
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Title/Link to more information |
Study Purpose |
Study Coordinator |
| Post Transplant 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. (Cytoxan) for GvHD Prophylaxis | The main purpose of this study is to assess the effects of cyclophosphamide (cytoxan) in the post transplant setting to prevent onset of acute graft-versus-host disease (GVHD) graft-versus-host disease (GVHD): Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the recipient's skin, intestines, or liver… . The primary objective is to determine the incidence of grade II-IV acute GVHD following Allogeneic (allo) Hematopoeitic Cell Transplant (HCT) using post-transplant cyclophosphamide (cytoxan) for patients with human leukocyte antigen human leukocyte antigen: (LEW-kuh-site ANT-i-jun) One of a group of proteins found on the surface of white blood cells and other cells. These antigens differ from person to person. A human leukocyte antigen test is done before a stem cell transplant to closely match a donor and a recipient. Also called HLA. (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors. |
This study is being conducted at UAB
Bone Marrow
Bone Marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
Transplantation and Cellular Therapy Program in Alabama. |
| Horse ATG in Patients With AA or Low/Int-1 Risk MDS | The goal of this clinical research clinical research: A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the… study is to learn if horse anti-thymocyte globulin (hATG), given in combination with methylprednisolone, cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. , and G-CSF (filgrastim or pegfilgrastim), can help to control AA and/or low-int-1 risk MDS. The safety of this drug combination will also be studied. |
This study is being conducted at MD Anderson Cancer Center |
| High-Tc Susceptometer to Monitor 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. | The purpose of this study is to validate a new suspectometer which uses very similar technology to a SQUID (Superconducting Quantum Interference Device) susceptometer. Investigators will do prospective, serial studies of the diagnosis and management of patients with iron overload, including thalassemia major (Cooley's anemia), sickle cell disease, aplastic anemia, myelodysplasia, hereditary hemochromatosis hemochromatosis: A condition that occurs when the body absorbs and stores too much iron. This leads to a condition called iron overload. In the United States, hemochromatosis is usually caused by a genetic disorder. Organ damage can occur if iron overload is not treated. , and other disorders |
This study is being conducted Columbia University Medical Center in New York City, NY |
Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 312 studies recruiting MDS patients in the United States.
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Title/Link to more information |
Study Purpose |
Study Coordinator |
| Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA (INSPIRE) | The primary objective of this study is to compare the overall survival (OS) of patients receiving intravenous (IV) rigosertib to the OS of patients receiving the physician's choice of treatment (PC) in a population of patients with myelodysplastic syndrome (MDS) after failure of treatment with a hypomethylating agent (HMA), azacitidine azacitidine: It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U… (AZA) or decitabine decitabine: It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Decitabine is approved by the U… (DEC). |
This study is being conducted in IL, MD, NY, and TX. Contact: Bernard Brownstein, MD Please refer to this study by its ClinicalTrials.gov identifier: NCT02562443 |
| Allo vs Hypomethylating/Best
Supportive Care
Supportive Care:
Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive…
in MDS (BMT CTN 1102) |
This study is designed as a multicenter trial, with biological assignment to one of two study arms; Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT), Arm 2: Non-Transplant Therapy/Best Supportive Care. |
This study is being conducted is currently being conducted at the Medical College of Wisconsin Contact: Heather Wittsack Contact: Adam Mendizabal, PhD |
| Sertraline in Treatment of Low-Risk Myelodysplastic Syndrome (SS1) |
This study will investigate the effects of sertraline in people with low-risk myelodysplastic syndrome (MDS). It is hoped that sertraline will decrease disease progression and reduce the need for blood transfusions blood transfusions: A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some… . |
This study is being conducted at the Baylor College of Medicine and the Michael E. DeBakey VA Medical Center in Houston, TX. Baylor College of Medicine Contact: Sarah McNees Michael E. DeBakey VA Medical Center Please refer to this study by its ClinicalTrials.gov identifier: NCT02452983 |
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.
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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
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| Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry | This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH). |
This study is being conducted at Johns Hopkins University Medical Center in Baltimore, MD and Roswell Park Cancer Institute in Buffalo, NY. |