Clinical Trials | Page 16 | 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.

Natural Killer (NK) Cells With HLA Compatible Hematopoietic Transplantation for High Risk Myeloid Malignancies

Status(es): Recruiting
Study Date(s): Saturday, June 1, 2013 to Friday, June 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 7 years to 65 years
The goal of this clinical research study is to find the highest tolerable dose of immune cells called natural killer (NK) cells that can be given with chemotherapy and a stem cell transplant to patients with AML and MDS. Researchers want to learn if adding NK cells will help make the stem cell transplant more effective in treating the disease. The safety of this treatment will also be studied. NK cells may kill cancer cells that remain in your body after your last chemotherapy treatment. The NK cells will be separated from blood from a relative of yours or from umbilical cord blood. These...

New York Blood Center National Cord Blood Program

Status(es): Recruiting
Study Date(s): Monday, September 13, 2010 to Tuesday, December 1, 2020
Disease(s): aplastic anemia
Age Group:
Umbilical cord blood donated to the New York Blood Center's National Cord Blood Program is collect, tested, processed, cryoprotected and frozen in liquid nitrogen for possible future transplantation to anyone who needs it. The Program has operated under a FDA IND exemption since 1996 and is licensed as a tissue bank by the New York State Department of Health. Candidates for transplant are patients who disease requires bone marrow transplantation but who do not have a suitable related bone marrow donor. Most patients are those with high risk of refractory leukemia, lymphoma, myelodysplasia,...

New York Blood Center National Cord Blood Program

Status(es): Recruiting
Study Date(s): Monday, February 1, 1993 to Tuesday, December 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: not provided
Umbilical cord blood is used as a source of hematopoietic stem cells for bone marrow reconstitution in patients who would be potential candidates for a bone marrow transplant from an unrelated marrow donor. The outcome of transplantation is obtained to assess cord blood myeloid and platelet engraftment, transplant related mortality, overall survival, graft vs. host disease and, for patients with leukemia, lymphoma or myelodysplasia, relapse.

Nivolumab and Ipilimumab With 5-azacitidine in Patients With Myelodysplastic Syndromes (MDS)

Status(es): Recruiting
Study Date(s): Tuesday, September 1, 2015 to Wednesday, September 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of this clinical research study is to learn if nivolumab and/or ipilimumab, with or without azacitidine, are safe to give to patients with MDS. Researchers also want to learn if the study drug combinations can help to control the disease.

Nivolumab in Acute Myeloid Leukemia (AML)

Status(es): Recruiting
Study Date(s): Wednesday, July 1, 2015 to Sunday, July 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The goal of this clinical research study is to find the highest tolerable dose of nivolumab that can be give in combination with idarubicin and cytarabine in patients with Myelodysplastic syndrome (MDS) and Acute Myeloid Leukemia (AML). The safety and effectiveness of this drug combination will also be studied.

Non-Myeloablative Conditioning for Unrelated Donor Umbilical Cord Blood Transplant

Status(es): Recruiting
Study Date(s): Wednesday, June 1, 2005 to Friday, June 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 75 years
RATIONALE: Drugs used in chemotherapy, such as fludarabine and cyclophosphamide, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Radiation therapy uses high-energy x-rays to kill cancer cells. An umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy and radiation therapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving sirolimus and mycophenolate mofetil after the transplant may stop this from...

Nonmyeloablative Allogeneic Transplant (Mini-allo)

Status(es): Recruiting
Study Date(s): Sunday, October 1, 2000 to Thursday, January 1, 2015
Disease(s): aplastic anemia, myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years to 72 years
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a nonmyeloablative preparative regimen.

Nonmyeloablative Haploidentical Transplant Followed by MLN9708

Status(es): Recruiting
Study Date(s): Monday, September 1, 2014 to Friday, December 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
In an attempt to reduce relapse risk and improve outcomes following haploidentical transplantation for patients with high risk hematologic malignancies, the investigators will implement several strategies to augment the well documented effect of NK cell alloreactivity seen in HLA-mismatched transplantation. These strategies include (1) choosing potential haploidentical donors for optimal NK-alloreactivity, (2) utilizing proteasome inhibition post-transplant with MLN9708 to both sensitize tumor cells to NK cytotoxicity and protect against graft-versus-host disease (GVHD), and (3) eliminating...

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Status(es): Recruiting
Study Date(s): Tuesday, April 1, 2014 to Sunday, August 1, 2021
Disease(s):
Age Group: 16 years to 80 years
Our ongoing nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplant protocol (03-H-0170) for patients with severe sickle cell disease (SCD) and B-thalassemia from HLA-matched family donors has excellent results thus far. Our long term leukocyte engraftment rate is 85-90% with the same disease-free survival. None of the engrafted patients had acute sickle-related events, significant toxicity associated with the conditioning regimen, or any evidence of graft versus host disease (GVHD). While these results rival the transplant outcomes from low risk transplant patients with B-...

Novel Approaches for Graft-versus-Host Disease Prevention Compared to Contemporary Controls (BMT CTN 1203)

Status(es): Recruiting
Study Date(s): Friday, August 1, 2014 to Wednesday, August 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 75 years
Acute Graft-versus-Host-Disease (GVHD) is an important cause of morbidity and mortality after allogeneic hematopoietic stem cell transplantation (HSCT). This study aims to determine if any of three new GVHD prophylaxis approaches improves the rate of GVHD and relapse free survival at one year after transplant compared to the current standard prophylaxis regimen.