Clinical Trials | Page 21 | 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:

Preventing Stem Cell Transplant Complications With a Blood Separator Machine

Status(es): Recruiting
Study Date(s): Wednesday, May 1, 2013 to Wednesday, February 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 2 years to 80 years
Background: - Researchers are working to make stem cell transplant procedures safer and more effective. One complication of transplants is graft-versus-host disease (GVHD). This complication happens when certain white blood cells from the donor attack the recipient's own body. Researchers want to test a blood separator machine that may help remove more of the donor's white blood cells before transplant. They will study donors and recipients during stem cell transplant to see how well this process can prevent GVHD and other complications. Objectives: - To see if a new blood separator machine...

Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation

Status(es): Recruiting
Study Date(s): Wednesday, February 26, 2014 to Saturday, February 1, 2020
Disease(s): aplastic anemia
Age Group: 1 year to 18 years
This is a Phase 2, open-label, randomized, controlled clinical study of pediatric subjects treated with pamidronate with calcium and vitamin D versus calcium and vitamin D alone following hematopoetic cell transplantation (HCT). The purpose of this study is to test the hypothesis that subjects receiving pamidronate with calcium and vitamin D will have higher lumbar spine bone mineral content (LBMC; adjusted for height, age, sex, Tanner stage, and race) measured by dual-energy X-ray tomography (DXA) at 1 year post-HCT than subjects receiving calcium and vitamin D alone (Control Group). Sixty...

PRO#1278: Fludarabine and Busulfan vs. Fludarabine, Busulfan and Total Body Irradiation (FLUBUTBI)

Status(es): Recruiting
Study Date(s): Tuesday, June 1, 2010 to Tuesday, December 1, 2015
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 to 70 years
This is a single institution study of fludarabine and busulfan versus fludarabine, busulfan and low dose total body irradiation in patients undergoing allogeneic stem cell transplantation. A study population of 80 subjects will be enrolled from The John Theurer Cancer Center at Hackensack University Medical Center. Subjects who are eligible to receive allogeneic hematopoietic stem cell transplantation according to the eligibility criteria will be consented and enrolled. Subjects will be randomly assigned to receive one of 2 conditioning regimen: fludarabine and busulfan, or fludarabine...

Radiation- and Alkylator-free Bone Marrow Transplantation Regimen for Patients With Dyskeratosis Congenita

Status(es): Recruiting
Study Date(s): Monday, August 6, 2012 to Sunday, October 1, 2017
Disease(s): aplastic anemia
Age Group: Up to 30 years
Dyskeratosis congenita (DC) is an inherited multisystem disorder, which classically presents with a clinical triad of skin pigment abnormalities, nail dystrophy, and oral leukoplakia. DC is part of a spectrum of telomere biology disorders, which include some forms of inherited idiopathic aplastic anemia, myelodysplastic syndrome, and pulmonary fibrosis and the congenital diseases Hoyeraal-Hreidarsson syndrome and Revesz syndrome. Progressive bone marrow failure (BMF) occurs in more than 80% of patients under 30 years of age and is the primary cause of morbidity and mortality, followed by...

Randomized Allogeneic Azacitidine Study

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2009 to Saturday, April 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 75 years
The goal of this clinical research study is to learn if Vidaza (azacitidine) will help to control the disease in patients with AML, CMML, or MDS after an allogeneic (donor) stem cell transplant. The safety of this drug will also be studied.

Randomized HaploCord Blood Transplantation vs. Double Umbilical Cord Blood Transplantation for Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Friday, April 29, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 year and older
The purpose of this study is compare the efficacy of haplo-cord transplant (investigational arm) with that of a more commonly used procedure in which only the cells contained in one or two umbilical cords are infused (standard arm). We hypothesize that reduced intensity conditioning and haplo-cord transplant results in fast engraftment of neutrophils and platelets, low incidences of acute and chronic graft versus host disease, low frequency of delayed opportunistic infections, reduced transfusion requirements, shortened length of hospital stay and promising long term outcomes. We also...

Randomized Trial of Lenalidomide, Bortezomib, Dexamethasone vs High-Dose Treatment With SCT in MM Patients up to Age 65 (DFCI 10-106)

Status(es): Recruiting
Study Date(s): Wednesday, December 1, 2010 to Saturday, September 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 to 65 years
The drugs, lenalidomide, bortezomib, and dexamethasone, are approved by the FDA. They have not been approved in the combination for multiple myeloma or any other type of cancer. Bortezomib is currently approved by the FDA for the treatment of multiple myeloma. Lenalidomide is approved for use with dexamethasone for patients with multiple myeloma who have received at least one prior therapy and for the treatment of certain types of myelodysplastic syndrome (another type of cancer affecting the blood). Dexamethasone is commonly used, either alone, or in combination with other drugs, to treat...

Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET)

Status(es): Recruiting
Study Date(s): Thursday, September 1, 2011 to Saturday, June 1, 2024
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This research is looking at two conditions, Essential Thrombocythemia (ET) and Polycythemia Vera (PV). ET causes people to produce too many blood cells called platelets and PV causes too many platelets and red blood cells to be made. Platelets are particles which circulate in the blood stream and normally prevent bleeding and bruising. Having too many platelets in the blood increases the risk of developing blood clots, which can result in life threatening events like heart attacks and strokes. When the number of red blood cells is increased in PV this will slow the speed of blood flow in the...

Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Wednesday, September 30, 2015 to Tuesday, September 1, 2020
Disease(s): aplastic anemia
Age Group: 18 years and older
PRIMARY OBJECTIVES: I. To demonstrate efficacy of this approach over the historical 2 step reduced intensity conditioning (RIC) approaches in the "vulnerable" population defined as: patients with hematopoietic cell transplant (HCT)-co-morbidity index (CI)/age scores >= 2, but no more than a score of 5 as based on the Sorror et al. data. SECONDARY OBJECTIVES: I. To compare the non-relapse mortality (NRM) and relapse related mortality (RRM) rates at 1 year for patients treated on this study to the that of patients undergoing haploidentical RIC hematopoietic stem cell transplantation (HSCT)...

Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Monday, June 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: up to 74 years
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of fludarabine, cyclophosphamide and low dose total body irradiation (TBI), is designed for the treatment of patients with advanced and/or high risk diseases.