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.

Personalized Natural Killer (NK) Cell Therapy in Cord Blood Transplantation (CBT)

Status(es): Recruiting
Study Date(s): Sunday, May 1, 2016 to Friday, May 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 1 to 60 years
The goal of this clinical research study is to learn if giving cells called natural killer (NK) cells after receiving 1 of 3 pre-treatment plans and a UCB transplant can improve response in participants with MDS, leukemia, lymphoma, or MM. The safety of this treatment and whether NK cells can lessen the risk of graft versus host disease (GVHD) will also be studied. If the disease is CD20 positive, participant will also receive rituximab on this study in addition to what is described above. CD20 is a type of marker for white blood cells. White blood cells help protect the body from infections...

Pharmacokinetic Guided Dose Escalation and Dose Confirmation With Oral Decitabine and Oral CDAi in Patients With MDS

Status(es): Recruiting
Study Date(s): Thursday, May 1, 2014 to Wednesday, June 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This 2-stage, open-label study will evaluate safety and pharmacokinetics of ASTX727, as well as determine the dose for the study's second stage. In the second stage the selected dose will be confirmed and evaluated for clinical activity, including response rate.

Phase 1 Study of TCP-ATRA for Adult Patients With AML and MDS (TCP-ATRA)

Status(es): Recruiting
Study Date(s): Sunday, February 1, 2015 to Friday, December 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Acute Myeloid Leukemia (AML) is a diverse disease that is fatal in the majority of patients. Acute promyelocytic leukemia (APL) however, a subtype of AML accounting for 5% of all cases, is very curable. APL cells are highly sensitive to the retinoid all-trans-retinoic acid (ATRA), which effectively differentiates the leukemic clone. Over 80% of APL patients can be cured with ATRA based therapies. For patients with non-APL AML, ATRA has little effect. Consequently, 85% of these patients will succumb to their disease despite conventional approaches. Little is known about mechanisms of...

Phase 1 Study to Evaluate MEDI4736 in MDS

Status(es): Recruiting
Study Date(s): Thursday, May 1, 2014 to Thursday, June 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 99 years
A dose-escalation and dose-expansion study of MEDI4736 (a monoclonal antibody that targets programmed cell death-1 ligand 1 [PD-L1]) to evaluate the safety, tolerability, PK, IM, and antitumor activity of MEDI4736 monotherapy and MEDI4736 in combination with azacitidine in adult patients with myelodysplastic syndrome.

Phase 1/2 Study of AG-221 in Subjects With Advanced Hematologic Malignancies With an IDH2 Mutation

Status(es): Recruiting
Study Date(s): Thursday, August 1, 2013 to Friday, September 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Study AG221-C-001 is a Phase 1/2, multicenter, open-label, dose-escalation, safety, PK/PD, and clinical activity evaluation of orally administered AG-221 in subjects with advanced hematologic malignancies that harbor an IDH2 mutation. The study includes a dose escalation phase to determine the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) and an expansion phase to further evaluate the safety, tolerability and clinical activity of AG-221 in select populations.

Phase 1/2 Study of the ERK1/2 Inhibitor BVD-523 in Patients With Acute Myelogenous Leukemia or Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Saturday, November 1, 2014 to Monday, May 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 100 years
This study is being performed to assess the safety, tolerability, and preliminary clinical effects of BVD-523 given orally, twice daily for 21-day cycles, in patients with Acute Myelogenous Leukemia (AML) orMyelodysplastic Syndrome (MDS).

Phase I Study of Velcade and Lenalidomide in Patients With Relapsed AML and MDS After Allogeneic Stem Cell Transplantation

Status(es): Recruiting
Study Date(s): Sunday, March 1, 2015 to Wednesday, November 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This research study is evaluating drugs called bortezomib and lenalidomide as a possible treatment formyelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). The purpose of this research study is to determine the safety and efficacy of the bortezomib and lenalidomide investigational combination. This drug combination has been used in the treatment of relapsed/refractory multiple myeloma and has been previously investigated in the treatment of MDS and AML, albeit at a lower dose of lenalidomide. In this research study, the investigators are looking for the highest dose of the...

Phase I, Dose Escalation Study of Decitabine

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Saturday, October 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 1 to 30 years
Decitabine is a hypomethylating agent that has shown significant anti-leukemic effect in Myelodysplastic Syndrome (MDS) and Acute Myeloblastic Leukemia (AML). This study is based on the hypothesis that Decitabine delivered after allo-hematopoietic stem cell transplant (HSCT) in patients with leukemia will enhance disease control by the allogeneic immune system and lead to a longer disease free survival. The study is designed to provide safety data of low-dosing in the post-transplant setting.

Phase I/II MAHCT w/ TCell Depleted Graft w/ Simultaneous Infusion Conventional and Regulatory T Cell

Status(es): Recruiting
Study Date(s): Thursday, December 1, 2011 to Thursday, December 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 13 years to 60 years
For patients with hematologic malignancies undergoing allogeneic myeloablative (MA) HCT with a T cell depleted graft, the infusion of naturally occurring regulatory T cells with conventional T cells (T cell addback) in pre-defined doses and ratios will reduce the incidence of acute graft vs host disease while augmenting the graft vs leukemia effect and improving immune reconstitution.

Phase I/II MEK162 Relapsed and/or Refractory Acute Myeloid Leukemia (AML) and Poor Prognosis, Not Suitable for or Unwilling to Receive Standard Therapy

Status(es): Recruiting
Study Date(s): Friday, August 1, 2014 to Wednesday, August 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of Phase 1 of this clinical research study is to find the highest tolerable dose of MEK162 that can be given to patients with relapsed and/or refractory AML, MDS, or ALL.