Clinical Trials | Page 18 | Aplastic Anemia and MDS International Foundation

Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

We are currently in the process of updating this section. For immediate information, please use the following links to view clinical trials listed on clinicaltrials.gov:

Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN 1203)

Status(es): Recruiting
Study Date(s): Friday, August 1, 2014 to Wednesday, August 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 75 years
Acute Graft-versus-Host-Disease (GVHD) is an important cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). This study aims to determine if any of three new GVHD prophylaxis approaches improves the rate of GVHD and relapse free survival at one year after transplant compared to the current standard prophylaxis regimen.

Omacetaxine and Decitabine in Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Saturday, May 2, 2015 to Tuesday, May 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 70 years and older
This clinical research study is made up of 2 phases. The goal of Phase 1 of the study is to test the safety of the combination of omacetaxine and decitabine and to find the best dose to give to future patients. The goal of Phase 2 of the study is to learn if this dose can help to control AML and/or MDS. The safety will then continue to be studied.

Omacetaxine in Patients With Intermediate-1 and Higher Risk Myelodysplastic Syndrome (MDS) Post Hypomethylating Agent (HMA) Failure

Status(es): Recruiting
Study Date(s): Friday, May 1, 2015 to Wednesday, May 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of this clinical research study is learn if omacetaxine can help to control MDS. The safety of this drug will also be studied.

ONC201 in Relapsed/Refractory Acute Leukemias and High-risk Myelodysplastic Syndromes (HR-MDS)

Status(es): Recruiting
Study Date(s): Sunday, November 1, 2015 to Tuesday, November 1, 2022
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of Phase I of this clinical research study is to find the highest tolerable dose of ONC201 that can be given to patients with relapsed or refractory AML, ALL, or MDS. The goal of Phase II of this study is to learn if the dose of ONC201 found in Phase I can help to control the disease. The safety of the study drug will be studied in both phases of this study. This is the first study using ONC201 in humans. ONC201 is in a very early stage of development for use in humans. Providing direct medical benefit to you is not the purpose of this study. While Phase II will look at the...

Organ-Sparing Marrow-Targeted Irradiation Before Stem Cell Transplant in Treating Patients With High-Risk Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Monday, June 1, 2015 to Wednesday, November 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 75 years
This pilot clinical trial aims to assess feasibility and tolerability of using an LINAC based "organ-sparing marrow-targeted irradiation" to condition patients with high-risk hematological malignancies who are otherwise ineligible to undergo myeloablative Total body irradiation (TBI)-based conditioning prior to allogeneic stem cell transplant. The target patient populations are those with ALL, AML, MDS who are either elderly (>50 years of age) but healthy, or younger patients with worse medical comorbidities (HCT-Specific Comorbidity Index Score (HCT-CI) > 4). The goal is to have the...

Outpatient Induction Chemotherapy in Treating Patients With Acute Myeloid Leukemia or Advanced Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Wednesday, May 1, 2013 to Saturday, October 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This pilot clinical trial studies the feasibility of having induction chemotherapy in an outpatient setting. Patients with acute leukemia (AML) or advanced myelodysplastic syndrome (MDS), at least 18 years of age will be examined. Treating eligible patients with induction chemotherapy in an outpatient setting may save in healthcare cost and improve a patients' quality of life.

Pacritinib in Combination With Low Dose Decitabine in Intermediate-High Risk Myelofibrosis or Myeloproliferative Neoplasm (MPN)/Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Friday, February 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
For the first 28 day cycle, all patients will be treated with single agent pacritinib at 200 mg twice daily. The investigators chose this starting dose based on the previous three phase I studies of pacritinib as a single agent which showed that the maximum tolerated dose (MTD) to be 500 mg, and subsequently, the dose of 400 mg daily was recommended for the phase II studies. Recently, the results of the phase III PERSIST-1 trial comparing pacritinib to best available therapy (BAT) in patients with MF was reported at the 2015 American Society of Clinical Oncology (ASCO) annual meeting....

Palifermin With Leuprolide Acetate for the Promotion of Immune Recovery Following Total Body Irradiation Based T-Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation

Status(es): Recruiting
Study Date(s): Saturday, December 1, 2012 to Friday, December 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 60 years
The purpose of this study is to help determine if palifermin and leuprolide acetate can help the immune system recover faster following a stem cell transplant. Blood stem cells are very young blood cells that grow in the body to become red or white blood cells or platelets. The transplant uses stem cells in the blood from another person. The donor can be a family member or a volunteer donor. This is called an allogeneic stem cell transplant. The investigators want to see if palifermin and leuprolide acetate can help the immune system recover faster after an allogenic transplant because...

Panobinostat With Fludarabine and Cytarabine for Treatment of Children With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Sunday, April 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 24 years
Cancer is the uncontrolled growth of human cells. The growth of normal human cells is controlled by multiple mechanisms. Panobinostat belongs to a class of chemotherapy drugs called "histone deacetylase (HDAC) inhibitors." HDAC inhibitors like panobinostat block enzymes known as histone deacetylases, which stops cancer cells from dividing and causes them to die. Fludarabine and cytarabine are chemotherapy drugs that are commonly used to treat pediatric patients with refractory or relapsed acute myeloid leukemia (AML) ormyelodysplastic syndrome (MDS). The purpose of this study is to test the...

Pasireotide in Prevention of GI Toxicity

Status(es): Recruiting
Study Date(s): Sunday, February 1, 2015 to Tuesday, January 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 and older
The purpose of this study is to evaluate if the drug, Pasireotide, is safe and effective in reducing the gastrointestinal side effects of the drugs received to prepare for allogeneic stem cell transplant. The study will also evaluate if Pasireotide is effective in reducing acute and chronic Graft-versus-Host-Disease (GvHD) after transplant.