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

Clinical Trials Report for February 2015

We have highlighted some clinical trials currently recruiting patients. Each has a specific purpose related to aplastic anemia, MDS, or PNH. Should you consider a clinical trial?  Ask your doctor or contact the study coordinators for further information. Learn more in the Clinical Trials section.

If you only want to see studies that are currently recruiting patients, select that option from the “recruitment” line at the beginning of the advanced search. To locate a clinical trial in your state, use the “advanced search” feature. Under locations, select your state(s) and your country.

Search for aplastic anemia studies actively recruiting patients. The following are a sample of the 67 open studies recruiting aplastic anemia patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of allogenic stem cell transplantation from either a matched unrelated donor or a mismatched reacted donor using the conditioning regimen of Cytoxan, reduced total body irradiation (TBI) and Campath IH. The principle measures of safety and efficacy will be :
1.    Patient survival probability at 100 days, 1 year and 2 years.
2.    Incidence of graft versus host disease (GVHD), as well as incidence of acute GVHD and chronic GVHD within 6 months and 2 years.
3.    Engraftment at 6 months, 1 year and 2 years
This study is being conducted at the Mayo Clinic, Rochester MN

Contact: Shakila Khan, MD    507-284-3442    Khan.shakila@mayo.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT00578266
Horse ATG in Patients With AA or Low/Int-1 Risk MDS The goal of this clinical research study is to learn if horse anti-thymocyte globulin (hATG), given in combination with methylprednisolone, cyclosporine, and G-CSF (filgrastim or pegfilgrastim), can help to control MM and/or low-int-1 risk MDS. The safety of this drug combination will also be studied.
hATG is made from horse blood and targets immune cells known as T-lymphocytes. Since T-lymphocytes are believed to be involved in causing low blood counts in AA and in some cases of MDS, killing these cells may help treat the disease.
Methylprednisolone and cyclosporine work to suppress immune cells called lymphocytes. This may help to improve low blood counts in AA and in some cases of MDS.
Filgrastim and pegfilgrastim are designed to cause white blood cells to grow. This may help to fight infections and help improve the white blood cell count.
This study is being conducted at University of Texas MD Anderson Cancer Center, Houston, Tx

Contact: Tapan Kadia, MD   713-563-3534
Eltrombopag for Moderate Aplastic Anemia The purpose of this study is to  evaluate the safety and effectiveness of eltrombopag in people with moderate
aplastic anemia who need treatment for significantly low blood cell counts.
Eltrombopag has been shown to safely increase platelet numbers in healthy volunteers and in patients with other chronic blood diseases, including severe aplastic anemia. Researchers are interested in looking at whether eltrombopag can be given to people with moderate aplastic anemia and significantly low blood cell counts.
This study is being conducted at the National Institutes of Health

Contact: Diane Madey, (301) 402-8282  madeydl@mail.nih.gov
Or Bogdan Dumitriu, M.D., (301) 339-4823 dumitriub@mail.nih.gov

Please refer to this study by its ClinicalTrials.gov identifier: NCT01328587

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 289 studies recruiting MDS patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

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 62 locations
Contact: Pfizer Clical Trial Call Center 1-800-718-1021
Please refer to this study by its ClinicalTrials.gov identifier: NCT01546038


Ipilimumab in Treating Patients With Relapsed or Refractory High-Risk Myelodysplastic Syndrome or Acute Myeloid Leukemia This phase I trial studies the side effects and best dose of ipilimumab in treating patients with high-risk myelodysplastic syndrome or acute myeloid leukemia that has come back or no longer responds to treatment. Monoclonal antibodies, such as ipilimumab, may interfere with the ability of cancer cells to grow and spread. This study is being conducted in
several states

Contact:
CT- Steven D. Gore    203-737-7103    steven.gore@yale.edu
MD- B. D. Smith    410-614-5068    bdsmith@jhmi.edu
NY-  Mark G. Frattini    212-851-4872    mgf2122@columbia.edu
NC- Matthew C. Foster    919-843-2447    matthew_foster@med.unc.edu
NC-  David A. Rizzieri    919-668-1040    david.rizzieri@dm.duke.edu
TX- Moshe Y. Levy    800-422-9567    mylevy@gmail.com  

Please refer to this study by its ClinicalTrials.gov identifier: NCT01757639
Phase 1 Study to Evaluate MEDI4736 This is a multicenter, open-label, Phase 1 study to assess the safety and antitumor activity of MEDI4736 in subjects with myelodysplastic syndrome. This study is being conducted in multiple sites.

Contact: AstraZeneca Clinical Study Information Center, 1-877-240-9479  information.center@astrazeneca.com

Please refer to this study by its ClinicalTrials.gov identifier: NCT02117219

Search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. The following are a sample of the 11 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

Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry

This study is a collection of data to evaluate safety and characterize progression of Paroxysmal Nocturnal Hemoglobinuria (PNH). Johns Hopkins University Medical Center , Baltimore
Contact: Lynn Sanders 203-439-9609 sandersl@alxn.com
Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360