Clinical Trials | Page 5 | 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.

Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia

Status(es): Recruiting
Study Date(s): Tuesday, February 2, 2010 to Wednesday, February 1, 2017
Disease(s): aplastic anemia
Age Group: Up to 60 years
The objective of this trial is to make an initial assessment for this new treatment regimen and to show it is equal or superior to the current standard practice. With this initial assessment be hope to gain information suggesting further study of this regimen or discontinuation of this regimen before exposing large numbers of patients to this new treatment option. We also will gain experience with this new regimen giving insights as to possible modifications in dosing and monitoring and selection of patients for future treatment in case of positive results. For this initial study we plan to...

Allogeneic Transplantation Using Timed Sequential Busulfan, Cladribine, and Fludarabine Conditioning in Patients With Acute Myelogenous Leukemia (AML) and Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Tuesday, October 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 2 years to 70 years
The goal of this clinical research study is to compare how well two different schedules of giving busulfan with fludarabine and cladribine before a stem cell transplant in patients with AML or MDS may help to control the disease. The safety of this drug combination will also be studied.

AML Therapy With Irradiated Allogeneic Cells

Status(es): Recruiting
Study Date(s): Saturday, February 1, 2014 to Tuesday, January 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This pilot clinical trial studies if cells donated by a close genetic relative can help keep acute myeloid leukemia (AML) from coming back after standard chemotherapy induces a complete remission. Blood cells obtained from a donor include a variety of blood cells that may trigger the patient's immune system to react against AML cells.

An Efficacy and Safety Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine in Participants With Higher-Risk Myelodysplastic Syndromes (HR MDS), Chronic Myelomonocytic Leukemia (CMML) and Low-Blast Acute Myelogenous Leukemia (AML)

Status(es): Recruiting
Study Date(s): Friday, January 1, 2016 to Friday, December 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this study is to evaluate the efficacy and safety of pevonedistat plus azacitidine versus single-agent azacitidine in participants with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia and low-blast acute myelogenous leukemia.

An Open-label Study of Lirilumab (BMS-986015) in Combination With 5-azacytidine (Vidaza) for the Treatment of Patients With Refractory/ Relapsed Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2015 to Wednesday, April 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of this clinical research study is to find the highest tolerated dose of the combination of lirilumab and 5-azacytidine that can be given to patients with AML or high-risk MDS. Researchers also want to learn if the drug combination can help to control the disease. The safety of the drug combination will also be studied.

An Open-Label, Dose-Escalation Study of INCB054329 in Patients With Advanced Malignancies

Status(es): Recruiting
Study Date(s): Friday, May 1, 2015 to Sunday, October 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a study of INCB054329 given to patients with advanced malignancies that will be conducted in three treatment groups. Each treatment group will have two parts; a dose escalation (Part 1) and an expansion (Part 2).

An Open-Label, Dose-Escalation/Dose-Expansion Safety Study of INCB059872 in Subjects With Advanced Malignancies

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Sunday, April 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an open-label, dose-escalation/dose-expansion study of INCB059872 in subjects with advanced malignancies. The study will be conducted in 2 parts. Part 1 (dose escalation) will determine the starting dose(s) of INCB059872 for dose expansion, based on maximum tolerated dose and/or a tolerated pharmacologically active dose. Part 2 (dose expansion) will further determine the safety, tolerability, efficacy, PK, and PD of the selected dose(s).

Azacitidine and Lenalidomide for Relapsed and Refractory Patients With Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Saturday, December 1, 2012 to Tuesday, November 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years or older
The primary objective of this study is to determine the complete remission/complete remission with incomplete recovery of blood counts (CR/CRi) rate for relapsed and refractory acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS) patients.

Azacitidine in Haploidentical Donor Hematopoietic Cell Transplantation

Status(es): Not yet recruiting
Study Date(s): Friday, July 1, 2016 to Thursday, November 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Allogeneic hematopoietic cell transplantation (allo-HCT) is a potentially curative therapy for patients with hematologic malignancies including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), and acute lymphoblastic leukemia (ALL); however, human leukocyte antigen (HLA)-matched donor availability continues to be a major hurdle. Historically, HLA haploidentical donor hematopoietic cell transplantation (haplo-HCT) was associated with high incidences of graft rejection and excessive non-relapse mortality (NRM), but recent advances utilizing post-transplant cyclophosphamide (PT-Cy...

Azacitidine in Patients Undergoing Matched Unrelated Stem Cell Transplantation

Status(es): Recruiting
Study Date(s): Monday, April 1, 2013 to Sunday, May 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 to 70 years
The purpose of this phase I/II study is to define the maximum tolerated dose of 5-AzaC and the effect on grade II-IV GvHD when given after matched unrelated donor transplant (MUD).