Clinical Trials | Page 4 | 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.

AC220 With 5-Aza or Low Dose Cytarabine

Status(es): Recruiting
Study Date(s): Friday, November 1, 2013 to Thursday, November 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of the Phase 1 part of this clinical research study is to find the highest tolerable dose of AC220 (quizartinib) that can be given with either 5-azacitidine (azacitidine) or cytarabine to patients with AML or MDS. The goal of the Phase 2 part of the study is to learn if quizartinib with either azacitidine or cytarabine can help to control AML or MDS. The safety of these combinations will also be studied.

Administration of Donor Multi TAA-Specific T Cells for AML or MDS (ADSPAM)

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Monday, April 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group:
This research study uses special blood cells called multiple tumor-associated antigen (TAA)-specific T cells (a new experimental therapy) to treat patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) which has come back, or may come back, or has not gone away after standard treatment, including an allogeneic hematopoietic stem cell transplant (HSCT). The investigators have previously used this sort of therapy to treat Hodgkin or non-Hodgkin lymphomas that are infected with Epstein-Barr virus (EBV), the virus that causes infectious mononucleosis ("mono" or the "kissing...

Adoptive Transfer of Haplo-identical DLI for AML and MDS

Status(es): Recruiting
Study Date(s): Tuesday, July 1, 2014 to Friday, July 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 55 years and older
The primary hypothesis is that chemotherapy followed by donor lymphocyte infusion (DLI) from HLA-haploidentical donors is a safe procedure that will not cause Graft versus Host Disease (GVHD) or increased treatment-related mortality. The Investigator further believes that this will improve outcomes of elderly patients with high-risk AML or MDS compared to chemotherapy alone, and that that this benefit will be even greater in donor-recipient pairs that share maternal-fetal microchimerism or non-inherited maternal antigen (NIMA) mismatch. A large part of this trial will include immune function...

Allo HSCT Using RIC for Hematological Diseases

Status(es): Recruiting
Study Date(s): Tuesday, March 1, 2016 to Wednesday, June 1, 2022
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 75 years
This is a phase II trial using a non-myeloablative cyclophosphamide/ fludarabine/total body irradiation (TBI) preparative regimen followed by a related or unrelated donor stem cell infusion. The primary objective is to evaluate rates of acute graft-versus-host disease (GVHD) grades II-IV and chronic GVHD with an updated GVHD prophylaxis of tacrolimus and mycophenolate mof

Allo vs Hypomethylating/Best Supportive Care in MDS (BMT CTN 1102)

Status(es): Recruiting
Study Date(s): Sunday, December 1, 2013 to Thursday, December 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 50 years to 75 years
This study is designed as a multicenter trial, with biological assignment to one of two study arms; Arm 1: Reduced intensity conditioning allogeneic hematopoietic cell transplantation (RIC-alloHCT), Arm 2: Non-Transplant Therapy/Best Supportive Care.

Allogeneic Bone Marrow Transplantation Using Less Intensive Therapy

Status(es): Recruiting
Study Date(s): Friday, March 1, 2002 to Thursday, December 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 75 years
RATIONALE: A peripheral stem cell transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy, or that have become cancer. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclophosphamide and fludarabine together with total-body irradiation followed by cyclosporine and mycophenolate mofetil before the transplant may stop this from happening. PURPOSE: This clinical trial is studying how well giving combination chemotherapy together with radiation therapy followed by...

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

Status(es): Recruiting
Study Date(s): Saturday, July 31, 2010 to Thursday, June 1, 2017
Disease(s): aplastic anemia
Age Group: 4 years old to 80 years old
Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS) associated with cytopenias. Patients with BMFS have traditionally been transplanted with bone marrow (BM) as a stem cell source. Although chronic graft versus host disease (cGVHD) occurs less commonly with BM compared to filgrastim (G-CSF) mobilized peripheral blood stem cell (PBSC) transplants, BM allografts have lower CD34+ progenitor cell...

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

Status(es): Recruiting
Study Date(s): Thursday, July 1, 2010 to Wednesday, June 14, 2017
Disease(s): aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 4 years to 80 years
Allogeneic hematopoietic stem cell transplantation (aHSCT) can cure patients with a variety of bone marrow failure syndromes (BMFS) including severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH) or myelodysplastic syndrome (MDS) associated with cytopenias. Patients with BMFS have traditionally been transplanted with bone marrow (BM) as a stem cell source. Although chronic graft versus host disease (cGVHD) occurs less commonly with BM compared to filgrastim (G-CSF) mobilized peripheral blood stem cell (PBSC) transplants, BM allografts have lower CD34+ progenitor cell...

Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies (AB-CliniMACs)

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Saturday, October 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: Up to 23 years
This is a single arm pilot study for patients using α/β T cell-depleted PSCT in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and GVHD, and one year leukemia free survival.

Allogeneic Stem Cell Transplant With Alpha/Beta T AND B Cell Depletion for Hematologic Malignancies (AB-CliniMACs)

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Tuesday, October 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 23 years
This is a single arm pilot study for patients using α/β T cell-depleted PSCT in with alternative donor sources with hematologic malignancies receiving alternative donor (unrelated or partially matched related) mobilized peripheral stem cells (PSCs) using the CliniMACS system for T cell depletion plus CD19+ B cell depletion to determine efficacy as determined by engraftment and GVHD, and one year leukemia free survival.