Clinical Trials | Page 19 | 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.

Phase I/II Trial to Investigate BI 836858 in Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Thursday, January 1, 2015 to Thursday, November 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Phase I: To investigate maximum tolerated dose (MTD), safety and tolerability, pharmacokinetics, exploratory biomarker and efficacy of BI 836858 monotherapy in patients with low or intermediate-1 riskmyelodysplastic syndromes (MDS) with symptomatic anemia. Phase II: To investigate safety and efficacy of BI 836858 plus Best Supportive Care compared to Best Supportive Care alone in low or intermediate-1 risk MDS patients with symptomatic anemia without a deletion 5q cytogenetic abnormality.

Phase I/II, Study of Selective Inhibitor of Nuclear Export (SINE) Selinexor (KPT-330) + Sorafenib in Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, December 1, 2015 to Sunday, December 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
There are 2 parts to this study: Part 1 (dose escalation) and Part 2 (dose expansion). The goal of Part 1 of this clinical research study is to find the highest tolerated dose of the combination of selinexor (KPT-330) and sorafenib (Nexavar) that can be given to patients with FLT3-ITD mutated AML or FLT3-mutated high-risk MDS. The goal of Part 2 of this study is to learn if the dose found in Part 1 can help to control the disease. The safety of the drug combination will also be studied in both parts of this study.

Phase II Decitabine (DAC) Versus Azacitidine (AZA) in Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Saturday, October 1, 2022
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of this clinical research study is to compare how 2 different drugs, decitabine and azacitidine, when given on a shorter than standard dosing schedule, may help to control MDS. The safety of each study drug given on these schedules will also be studied.

Phase II Study of 5-azacytidine Maintenance After Transplant for AML or MDS (UPCI 13-165)

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2015 to Wednesday, August 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Despite improvements in outcomes after Hematopoietic Cell Transplantation (HCT) for Acute Myeloid Leukemia (AML) and Myelodysplastic Syndrome (MDS), the risk of relapse remains high and is the most common cause of mortality after HCT. Moreover, treatment options for relapse after HCT are limited. Strategies to reduce relapse with maintenance therapy in patients who are at high risk are needed to improve survival. 5-aza is a hypomethylating agent that has shown immune modulating properties that may enhance the graft-versus-leukemia (GVL) effect, including upregulation of tumor-associated...

Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, May 25, 2015 to Saturday, July 1, 2017
Disease(s): aplastic anemia
Age Group: 18 years and older
This will be an open-label, single arm, phase IIa study in subjects with AA or Hypoplastic MDS. Eligible subjects will receive subcutaneous (SC) injections of BL-8040 monotherapy over 10 days. From Day 11 through Day 14, subjects will receive hATG, Methylprednisolone and Cyclosporine. From Day 15 until Day 30 (of the first month), subjects will continue treatment only with Methylprednisolone and Cyclosporine. Cyclosporine will continue daily through Month 6/Day 30 (M6/D30) (end of study treatment). Beginning on M2/D1, BL-8040 will be administered daily as part of the maintenance period for...

Pilot Study of Lupron to Improve Immune Function After Allogeneic Bone Marrow Transplantation

Status(es): Recruiting
Study Date(s): Tuesday, March 1, 2011 to Saturday, April 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 9 years to 40 years
Background: One way to treat certain cancers of the blood and immune system is to give a patient stem cells from the bone marrow of a donor whose genes are very similar but not identical to the patient s. One problem with these transplants is that the new immune cells may not work as well in the recipient as they did in the donor. The result may be that the immune system will not work as well. This can increase the risk of severe infections and other complications. Researchers are studying the use of drugs that lower hormone levels and may allow the immune system to recover in a way that...

Pilot Study to Assess Hematologic Response in Patients With Acute Myeloid Leukemia or High Risk Myelodysplastic Syndromes Undergoing Monotherapy With Exjade (Deferasirox)

Status(es): Recruiting
Study Date(s): Friday, August 1, 2014 to Monday, August 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this trial is to examine the hematologic response rate of Exjade® in patients with AML and high risk MDS and chronic iron overload from blood transfusions. Deferasirox has been developed as an iron-chelating agent, and unlike deferoxamine, a previously developed iron chelator, deferasirox has the advantage of oral administration.

Pomalidomide After Combination Chemotherapy in Treating Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Sunday, December 1, 2013 to Thursday, September 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 65 years
This phase I trial studies the side effects and best dose of pomalidomide after combination chemotherapy in treating patients with newly diagnosed acute myeloid leukemia or high-risk myelodysplastic syndrome. Drugs used in chemotherapy, such as cytarabine, daunorubicin hydrochloride, and etoposide, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Pomalidomide may kill cancer cells by stopping blood flow to the cancer and by stimulating white blood cells to kill cancer cells. Giving more...

Post Transplant Cyclophosphamide (Cytoxan) for GvHD Prophylaxis

Status(es): Recruiting
Study Date(s): Sunday, November 3, 2013 to Sunday, July 1, 2018
Disease(s): aplastic anemia
Age Group: 19 years to 65 years
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. Other objectives for this study will be the determination of disease-free survival (DFS) and overall survival (OS)...

Preventing Stem Cell Transplant Complications With a Blood Separator Machine

Status(es): Recruiting
Study Date(s): Wednesday, May 1, 2013 to Wednesday, February 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 2 years to 80 years
Background: - Researchers are working to make stem cell transplant procedures safer and more effective. One complication of transplants is graft-versus-host disease (GVHD). This complication happens when certain white blood cells from the donor attack the recipient's own body. Researchers want to test a blood separator machine that may help remove more of the donor's white blood cells before transplant. They will study donors and recipients during stem cell transplant to see how well this process can prevent GVHD and other complications. Objectives: - To see if a new blood separator machine...