Clinical Trials | Page 14 | 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.

We are currently in the process of updating this section. For immediate information, please use the following links to view clinical trials listed on clinicaltrials.gov:

HSCT for Patients With Fanconi Anemia Using Risk-Adjusted Chemotherapy (RAFA)

Status(es): Recruiting
Study Date(s): Tuesday, April 1, 2014 to Wednesday, May 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 3 months and older
The purpose of this study is to determine whether the use of lower doses of busulfan and the elimination of cyclosporine will further reduce transplant-related side effects for patients with Fanconi Anemia (FA). Patients will undergo a transplant utilizing mis-matched related or matched unrelated donors following a preparative regimen of busulfan, fludarabine, anti-thymocyte globulin and cyclophosphamide.

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Status(es): Recruiting
Study Date(s): Monday, April 1, 2013 to Saturday, December 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 55 years
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Status(es): Recruiting
Study Date(s): Wednesday, April 25, 2012 to Saturday, December 1, 2018
Disease(s): aplastic anemia
Age Group: Up to 55 years
The purpose of this clinical trial is to investigate the safety of human placental-derived stem cells (HPDSC) given in conjunction with umbilical cord blood (UCB) stem cells in patients with various malignant or nonmalignant disorders who require a stem cell transplant. Patients will get either full dose (high-intensity) or lower dose (low intensity) chemo- and immunotherapy followed by a stem cell transplantation with UCB and HPDSC.

Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome

Status(es): Not yet recruiting
Study Date(s): Tuesday, September 1, 2015 to Tuesday, November 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase Ib trial studies the side effects and best dose of ibrutinib when given together with azacitidine in treating patients with myelodysplastic syndrome that is likely to occur or spread (higher risk) and who were previously treated or untreated and unfit for or refused intense therapy. Ibrutinib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

IL-15 Super Agonist ALT-803 to Treat Relapse Of Hematologic Malignancy After Allogeneic SCT

Status(es): Recruiting
Study Date(s): Friday, November 1, 2013 to Thursday, June 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a multi-center, phase I/II clinical trial for patients who have relapsed more than 60 day after allogeneic transplant for a hematologic malignancy. The study consists of two phases. The dose finding phase is a modified version of a phase I trial and the extended phase is a modified version of a phase II trial. The primary objective of the dose finding phase is to determine the maximum tolerated, minimum efficacious dose (MTD/MED) of a interleukin-15 (IL-15) super agonist complex (ALT-803) when given once weekly for 4 weeks in the outpatient setting. The study will follow a standard 3...

Immunochemotherapy and AlloSCT in Patients With High Risk CD33+ AML/MDS

Status(es): Recruiting
Study Date(s): Tuesday, November 1, 2011 to Friday, December 1, 2017
Disease(s):
Age Group: up to 30 years
Targeted immune therapy with gemtuzumab ozogamicin (Mylotarg) in combination with chemotherapy followed by allogeneic stem cell transplantation will be given to patients with high risk acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS).

Inducible Regulatory T Cells (iTregs) in Non-Myeloablative Sibling Donor Peripheral Blood Stem Cell Transplantation

Status(es): Recruiting
Study Date(s): Friday, November 1, 2013 to Thursday, December 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 75 years
This is a phase I single center dose escalation study with an extension at the best available dose to determine the tolerability of inducible regulatory T cells (iTregs) when given to adult patients undergoing non-myeloablative HLA-identical sibling donor peripheral blood stem cell (PBSC) transplantation for the treatment of a high risk malignancy. Up to 5 dose cohorts will be tested. Once the tolerable dose is determined for iTregs, enrollment will continue with an additional 10 patients using sirolimus/Mycophenolate mofetil (MMF) graft-versus-host disease (GVHD) prophylaxis to gain further...

Intensity Modulated Total Marrow Irradiation, Fludarabine Phosphate, and Melphalan in Treating Patients With Relapsed Hematologic Cancers Undergoing a Second Donor Stem Cell Transplant

Status(es): Recruiting
Study Date(s): Monday, December 1, 2014 to Wednesday, November 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group:
This phase I trial studies the side effects and the best dose of intensity modulated total marrow irradiation (IMTMI) when given together with fludarabine phosphate and melphalan in treating patients with cancers of the blood (hematologic) that have returned after a period of improvement (relapsed) undergoing a second donor stem cell transplant. IMTMI is a type of radiation therapy to the bone marrow that may be less toxic and may also reduce the chances of cancer to return. Giving fludarabine phosphate, melphalan, and IMTMI before a donor stem cell transplant may help stop the growth of...

Intra-Osseous Co-Transplant of UCB and hMSC

Status(es): Recruiting
Study Date(s): Wednesday, July 1, 2015 to Wednesday, March 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 to 75 years
This clinical trial studies intra-osseous donor umbilical cord blood and mesenchymal stromal cell co-transplant in treating patients with hematologic malignancies. Giving low doses of chemotherapy and total-body irradiation before a co-transplant of donor umbilical cord blood and mesenchymal stromal cells into the bone (intra-osseous) helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect)....

Iodine I 131 Monoclonal Antibody BC8, Fludarabine Phosphate, Cyclophosphamide, Total-Body Irradiation and Donor Bone Marrow Transplant in Treating Patients With Advanced Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, or High-Risk Myelodysplastic Sy

Status(es): Recruiting
Study Date(s): Monday, October 1, 2007 to Thursday, September 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase II trial studies the side effects and best dose of iodine I 131monoclonal antibody BC8 when given together with fludarabine phosphate, cyclophosphamide, total-body irradiation, and donor bone marrow transplant, and to see how well they work in treating patients with acute myeloid leukemia or acute lymphoblastic leukemia that has spread to nearby or other places in the body (advanced), or high-riskmyelodysplastic syndrome. Giving chemotherapy drugs, such as fludarabine phosphate and cyclophosphamide, and total-body irradiation before a donor bone marrow transplant helps stop the...