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

Clinical Trials Report for July 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 79 open studies recruiting aplastic anemia patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

Reduced Intensity Conditioning Using CD3+/CD19+ Depletion for Non Malignant Transplantable Diseases This is a Phase II trial to determine the ability of a reduced intensity conditioning regimen to allow successful engraftment with CD3+ /CD19+ depleted peripheral stem cell grafts from mismatched donors. There are two conditioning regimens depending upon patient diagnosis and age.

This study is being conducted at the Children’s Hospital of Philadelphia, PA

Contact: Margaret Tartaglione, RN, 215-590-4029 or tartaglione@email.chop.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT02277639
Genetic Variation and Variability in Posaconazole Pharmacokinetics in Children The main goal of this study is to see how the body breaks down an antifungal drug named posaconazole in children with certain cancers, blood disorders, or transplantation of bone marrow or similar blood cells. This study will also help us learn whether a child's age, genetics, or disease affect how well the body breaks down posaconazole. This study is being conducted at Children’s Mercy Hospital in Kansas City, Missouri.

Contact: Joanne L Thurber, RN, CCRC
816-234-3076 jthurber@cmh.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT02358499

Seach for myelodysplastic syndromes studies actively recruiting patients. The following MDS studies are a sample of the 315 studies recruiting MDS patients in the United States.

Title/Link to more information

Study Purpose

Study Coordinator

The Myelodysplasia Transplantation-Associated Outcomes (MDS-TAO) Study

The purpose of this observational study is to compare overall survival in older adults with myelodysplastic syndromes (MDS) who receive reduced intensity conditioning hematopoietic stem cell transplant (RIC HSCT) versus those who do not receive HSCT. This study is being conducted in Boston, MA.

Beth Israel Deaconess Medical Center—contact David Avigan, MD 616-667-9920 davigan@bidmc.harvard.edu

Dana Farber Cancer Institute—contact Gregory Abel, MD, MPH 717-632-2304 GAABEL@PARTNERS.ORG 

Massachusetts General Hospital—contact Yi-Ben Chen, MD 617-726-1124 yachen@partners.org

Please refer to this study by its ClinicalTrials.gov identifier: NCT02390414

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 Florida, Maryland, Massachusetts, New York, Ohio, and Texas

Contact: Guillermo Garcia-Manero, MD 713-745-3428

Please refer to this study by its ClinicalTrials.gov identifier: NCT02378701

GVHD Prophylaxis With Post Transplant Cyclophosphamide for Patients With Renal Insufficiency Undergoing a Conventional 10/10 HLA-matched Related or Unrelated Donor Allogeneic Hematopoietic Stem Cell Transplant


This is a pilot study which will be done in a small number of patients. The purpose of this study is to test the safety and benefit of giving a type of chemotherapy - cyclophosphamide - after the transplant to prevent graft versus host disease (GVHD) in patients with abnormal kidney function. GVHD is one of the most common complications of a stem cell transplant.

This study is being conducted in NY.

Contact: Ann Jakubowski, Ph.D., M.D.
212-639-5013 or

Esperanza Papadopoulos, M.D
212-639-8276    

Please refer to this study by its ClinicalTrials.gov identifier: NCT02360111

 

Please search for a list of current paroxysmal nocturnal hemoglobinuria studies actively recruiting patients. The following are a recent sample of the 9 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