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

Clinical Trials Report for April 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.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.

Title/Link to more information

Study Purpose

Study Coordinator

Post Transplant Cyclophosphamide (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). 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 (HLA) matched unrelated (MUD) and mismatched unrelated (MMUD) donors.

This study is being conducted at UAB Bone Marrow Transplantation and Cellular Therapy Program in Alabama.

Contact: Lisa D Williams, RN    
205-934-0066    
liwilliams@uabmc.edu 

Contact: Ashleigh Brooks, RN    
205-996-8023    
kathrynbrooks@uabmc.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT02065154

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 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
Contact: Tapan Kadia, MD    713-563-3534    
        
Please refer to this study by its ClinicalTrials.gov identifier: NCT01624805

High-Tc Susceptometer to Monitor Transfusional Iron Overload 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, and other disorders

This study is being conducted Columbia University Medical Center in New York City, NY

Contact: Gary Brittenham, MD
212 305-7005
gmb31@columbia.edu

Contact: Sujit Sheth, M.D
212 746-3439
shethsu@med.cornell.edu

Please refer to this study by its ClinicalTrials.gov identifier: NCT01241357

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.

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 (AZA) or decitabine (DEC).

This study is being conducted in IL, MD, NY, and TX.

Contact: Bernard Brownstein, MD    
(267) 759-3676
bbrownstein@onconova.us    

Please refer to this study by its ClinicalTrials.gov identifier: NCT02562443

Allo vs Hypomethylating/Best Supportive Care 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
hwittsack@emmes.com

Contact: Adam Mendizabal, PhD
amendizabal@emmes.com
    
Please refer to this study by its ClinicalTrials.gov identifier: NCT02016781

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.

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: Gustavo Rivero, MD
713-798-5103
garivero@bcm.edu   

Contact: Sarah McNees
713-798-8810
smcnees@bcm.edu  

Michael E. DeBakey VA Medical Center
Contact: Gustavo A Rivero, MD
713-794-7134
gustavo.rivero@va.gov   

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.

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).

This study is being conducted at Johns Hopkins University Medical Center in Baltimore, MD and Roswell Park Cancer Institute in Buffalo, NY.

Contact: Lynn Sanders 203-439-9609
sandersl@alxn.com

Please refer to this study by its ClinicalTrials.gov identifier: NCT01374360