Clinical Trials | Page 9 | 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:

Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease

Status(es): Recruiting
Study Date(s): Sunday, February 1, 2015 to Thursday, February 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 to 65 years
This pilot trial offers the unique opportunity for both the treatment of multiple myeloma or other hematologic disorder for which hematopoietic stem cell transplantation would be ordinarily indicated and the reversal of end-stage renal failure, while avoiding the risks associated with long-term standard anti-rejection therapy used in renal transplantation. The primary objectives of this study are to assess renal allograft tolerance (that is, the acceptance of the kidney without the need for anti-rejection therapy), assess anti-tumor response rates in multiple myeloma and other malignancies,...

Conditioning Regimen and the Transplantation of Unrelated Donor Umbilical Cord Blood in Patients With Hematologic Malignancies.

Status(es): Recruiting
Study Date(s): Friday, August 1, 2008 to Tuesday, August 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 70 years
The traditional way of doing a donor transplant is to give high doses of chemotherapy and radiation before giving the stem cells. However, high doses of chemotherapy and radiation can have serious side-effects. The doctors think that the transplant will be safer and more likely to be successful with reduced doses of chemotherapy and radiation. The purpose of this study is to find out how good a combination of chemotherapy and radiation at reduced doses followed by a cord blood transplant are at treating cancer. The stem cells chosen for the transplant are from umbilical cord blood. Umbilical...

Controlled Study of Rigosertib Versus Physician's Choice of Treatment in MDS Patients After Failure of an HMA (INSPIRE)

Status(es): Recruiting
Study Date(s): Friday, October 2, 2015 to Friday, June 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 79 years
The primary objective of this study is to compare the overall survival (OS) of patients receiving intravenous (IV) rigosertib to the OS of patients receiving the physician's choice of treatment (PC) in a population of patients with myelodysplastic syndrome (MDS) after failure of treatment with a hypomethylating agent (HMA), azacitidine (AZA) or decitabine (DEC).

Cord Blood Fucosylation to Enhance Homing and Engraftment in Patients With Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Sunday, July 1, 2012 to Sunday, July 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 1 to 80 years
The goal of this clinical research study is to learn if it is safe and feasible to transplant changed cord blood for patients with leukemia or lymphoma. Researchers also want to learn if this can help to control the disease. The cord blood will be changed to make use of sugar that is found in small amounts in blood cells. It plays a role in signaling where in the body the transplanted cells should go to. Adding more sugars to the cord blood cells in the laboratory is designed to help the cord blood cells find their way faster to the bone marrow. This may help your blood counts to recover...

Cord Blood Transplant

Status(es): Recruiting
Study Date(s): Friday, January 11, 2013 to Friday, December 1, 2017
Disease(s): aplastic anemia
Age Group: 16 years to 70 years
Allogeneic hematopoietic cell transplantation (allo- HCT) is a curative therapy for the treatment of hematological and non-hematological malignancies and certain non-malignant conditions. Bone marrow or peripheral blood from a Human Leukocyte Antigen (HLA) matched sibling donor is the most commonly used source of allogeneic stem cells. However, HLA matched siblings are available for less than one third of the patients who require allo- SCT. In the absence of an HLA matched sibling, volunteer unrelated donors or partially mismatched related donors (PMRD), stored cord blood may be used as a...

CPI-613 in Treating Patients With Myelodysplastic Syndromes Who Failed Previous Therapy

Status(es): Recruiting
Study Date(s): Thursday, August 1, 2013 to Monday, August 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This pilot clinical trial studies 6, 8-bis (benzylthio) octanoic acid (CPI-613) in treating patients withmyelodysplastic syndromes who failed previous therapy. Sometimes when chemotherapy or biological therapy is given, it does not stop the growth of tumor cells. The tumor is said to be resistant to treatment. 6, 8-bis (benzylthio) octanoic acid may interfere with the growth of tumor cells and may be an effective treatment formyelodysplastic syndromes that did not respond to previous therapy.

Crenolanib in Combination With Sorafenib in Patients With Refractory or Relapsed Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Monday, June 6, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 1 to 39 years
This is a pilot study to characterize the toxicity profile, to determine the maximum tolerated dose of the combination of crenolanib and sorafenib, and to determine the feasibility of administering these drugs in patients with relapsed or refractory hematologic malignancies, including acute myeloid leukemia (AML), AML with prior myelodysplastic syndrome (MDS), and myeloperoxidase (MPO)-positive mixed phenotype acute leukemia with FLT3-internal tandem duplication (ITD) and tyrosine kinase domain (TKD) mutations. The study will include two phases: The dose-escalation phase will characterize...

Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia

Status(es): Recruiting
Study Date(s): Friday, March 5, 2010 to Wednesday, June 1, 2016
Disease(s):
Age Group: Up to 59 years
OBJECTIVES: Primary To determine the probability of engraftment in patients with Fanconi anemia treated with cyclophosphamide, fludarabine phosphate, and antithymocyte globulin followed by HLA-genotypically identical sibling donor hematopoietic stem cell transplantation that is T-cell depleted. Secondary To evaluate the incidence of acute graft-versus-host disease (GVHD) and chronic GVHD in patients treated with this regimen. To evaluate the incidence of regimen-related toxicity in these patients. To evaluate the 1-year survival of patients treated with this regimen. To evaluate the...

Dasatinib for Immune Modulation After Donor Stem Cell Transplant for Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Tuesday, May 1, 2012 to Saturday, October 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This study uses a drug called dasatinib to produce an anti-cancer effect called large granular lymphocyte cellular expansion. Large granular lymphocytes are blood cells known as natural killer cells that remove cancer cells. Researchers think that dasatinib may cause large granular lymphocyte expansion to happen in patients who have received a blood stem cell transplant (SCT) between 3 to 15 months after the SCT. In this research study, researchers want to find how well dasatinib can be tolerated, the best dose to take of dasatinib and how to estimate how often large granular lymphocytic...

Decitabine and Cytarabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia, High Risk Myelodysplastic Syndrome, or Myeloproliferative Neoplasm

Status(es): Recruiting
Study Date(s): Sunday, June 1, 2014 to Wednesday, March 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 60 years and older
This clinical trial studies decitabine and cytarabine in treating older patients with newly diagnosed acute myeloid leukemia, myelodysplastic syndrome that is likely to come back or spread to other places in the body, or myeloproliferative neoplasm. Drugs used in chemotherapy, such as decitabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving decitabine and cytarabine may work better than standard therapies in treating cancers of the bone marrow and blood cells, such as acute myeloid leukemia, ...