Clinical Trials | Page 8 | Aplastic Anemia & 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.

An European Platform for Translational Research in Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Monday, September 30, 2019 to Friday, September 30, 2022
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Rationale Myelodysplastic syndromes (MDS) are rare cancers with unmet medical needs. Study of MDS has been rapidly transformed by genome characterization. The investigators hypothesize that comprehensive analyses of large patient population will allow to correctly estimate the effect of each mutation on clinical outcomes, and that niche factors and immune dysfunctions may influence the development of MDS, clonal evolution and response to treatments

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.

APL-2 Long Term Safety and Efficacy Extension Study

Status(es): Active, not recruiting
Study Date(s): Tuesday, August 28, 2018 to Saturday, August 20, 2022
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This is an Open Label, Non-Randomized, Multi-Center Extension Study. Eligible subjects will have previously completed an APL-2 study.

APR-246 in Combination With Azacitidine for TP53 Mutated AML (Acute Myeloid Leukemia) or MDS (Myelodysplastic Syndromes) Following Allogeneic Stem Cell Transplant

Status(es): - Unknown -
Study Date(s): Tuesday, April 30, 2019 to Friday, October 1, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
A multi-center, open label, Phase II clinical trial to assess the safety and efficacy of APR-246 in combination with azacitidine as maintenance therapy after allogeneic HSCT (hematopoietic stem cell transplant) for patients with TP53 mutant AML or MDS. Detailed Description: A multi-center, open label, Phase II clinical trial to assess the safety and efficacy of APR-246 in combination with azacitidine as maintenance therapy after allogeneic HSCT for patients with TP53 mutant AML or MDS. Patients will be prescreened for TP53 mutant AML or MDS before they have a HSCT. In order to proceed with...

ASTX727 and FT-2102 in Treating IDH1-Mutated Recurrent/Refractory Myelodysplastic Syndrome or Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Thursday, August 22, 2019 to Thursday, March 31, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase Ib/II trial studies the side effects and best dose of FT-2102 when given together with ASTX727 in treating patients with IDH1-mutated myelodysplastic syndrome or acute myeloid leukemia that has come back (recurrent) or does not respond to treatment (refractory). ASTX727 is an oral deoxyribonucleic acid (DNA) methyltransferase (DNMT) inhibitor. DNA methylation is necessary for cell differentiation and development. Changes to the methylation profile can lead to DNA instability which can cause diseases like cancer. DNMT inhibitors target and inhibit these changes. FT-2102 is an...

ATG Plus PTCy vs ATG for CGVHD Prophylaxis

Status(es): Not yet recruiting
Study Date(s): Saturday, February 1, 2020 to Wednesday, June 30, 2021
Disease(s): graft versus host disease (GVHD), myelodysplastic syndromes (MDS)
Age Group: 16 years to 70 years
A Randomized Pilot Trial to test the feasibility of comparing anti-thymocyte globulin plus post transplant cyclophosphamide with anti-thymocyte globulin alone to prevent chronic graft versus host disease.

Augmentation of the Graft vs. Leukemia Effect Via Checkpoint Blockade With Pembrolizumab

Status(es): Recruiting
Study Date(s): Tuesday, April 18, 2017 to Saturday, October 31, 2020
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years or older
This is a single arm, open-label, Phase 1b study of pembrolizumab for patients with myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), and acute lymphoblastic leukemia (ALL) whose disease has relapsed after receiving allogeneic hematopoetic stem cell transplant.

Azacitidine and Enasidenib in Treating Patients With IDH2-Mutant Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Tuesday, December 26, 2017 to Tuesday, February 28, 2023
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 12 years and older
This phase II trial studies the side effects and how well azacitidine and enasidenib work in treating patients with IDH2-mutant myelodysplastic syndrome. Azacitidine and enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Azacitidine Plus Nivolumab Following Reduced-intensity Allogeneic PBSC Transplantation for Patients With AML and High-risk Myelodysplasia

Status(es): Recruiting
Study Date(s): Thursday, October 10, 2019 to Monday, October 31, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a phase I clinical trial that will define the maximum tolerated dose (MTD) and investigate the feasibility and safety of the combination of nivolumab and azacitidine after reduced-intensity allogeneic PBSC transplantation. Dose escalation will follow a traditional 3+3 design. The investigators will first escalate the dose of single agent nivolumab to determine its MTD (if any, at the doses tested), with an expanded cohort at the MTD or highest dose tested. The investigators will then combine escalating azacitidine in combination of with nivolumab at its determined MTD or highest dose...

Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Tuesday, December 17, 2019 to Thursday, September 1, 2022
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase I/II trial studies the side effects and best dose of gilteritinib and to see how well it works in combination with azacitidine and venetoclax in treating patients with FLT3-mutation positive acute myeloid leukemia or high-risk myelodysplastic syndrome that has come back (recurrent) or has not responded to treatment (refractory). Drugs used in chemotherapy, such as azacitidine and venetoclax, 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. Gilteritinib may stop the growth of...