Clinical Trials | Page 7 | Aplastic Anemia & 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.

A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab (PNH)

Status(es): Active, not recruiting
Study Date(s): Tuesday, May 8, 2018 to Thursday, December 31, 2020
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years to 65 years
To determine the effectiveness of ACH-0144471 in improving anemia when given with eculizumab for 24 weeks in patients with PNH.

A Trial to Evaluate Two Schedules of MS275 in Combination With 5AC in Elderly Patients With Acute Myeloid Leukemia (AML)

Status(es): Recruiting
Study Date(s): Friday, July 1, 2011 to Wednesday, June 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 60 and older
This research is being done to help us learn how to best use new drugs which may be active against acute myeloid leukemia (AML). Two study drugs will be tested: 5AC (5-azacitidine) and entinostat. 5AC improves blood counts in 50 - 60% of patients with MDS and has also shown promise in AML. Entinostat has undergone early testing in patients with MDS and AML. It has decreased the blast count in some patients' blood and bone marrow and has improved the blood counts in some patients. The combinations of these two classes of drugs are well tolerated and appear to work well together in laboratory...

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

Status(es): Recruiting
Study Date(s): Wednesday, February 17, 2016 to Friday, February 28, 2025
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: All ages
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). EBV is found in cancer cells of up to half of all patients with...

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

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): Friday, February 4, 2011 to Wednesday, June 30, 2021
Disease(s): aplastic anemia, graft versus host disease (GVHD), myelodysplastic syndromes (MDS), paroxysmal nocturnal hemoglobinuria (PNH), pure red cell aplasia (PRCA)
Age Group: 4 years to 80 years
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient. Because stem cell transplants can have serious complications, researchers are interested in developing new approaches to stem cell transplants that will reduce the likelihood of these complications. By reducing the number of white blood cells included in the blood taken during the stem 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.

ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status(es): Active, not recruiting
Study Date(s): Tuesday, December 20, 2016 to Sunday, January 1, 2023
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who had never been treated with a complement inhibitor (treatment-naïve).

ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

Status(es): Active, not recruiting
Study Date(s): Monday, June 5, 2017 to Monday, March 1, 2021
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The primary purpose of this study was to assess the noninferiority of ravulizumab compared to eculizumab in adult participants with PNH who were clinically stable after having been treated with eculizumab for at least 6 months.

AML/MDS Drug Sensitization by in Vivo Chemotherapy Administration

Status(es): Not yet recruiting
Study Date(s): Friday, February 28, 2020 to Friday, February 28, 2025
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
In this study, the investigators will explore the feasibility of ex vivo drug screening to predict sensitivity to chemotherapy resistance and to identify novel synergy between chemotherapies.

An Efficacy and Safety Study of Luspatercept (ACE-536) for the Treatment of Anemia Due to IPSS-R Very Low, Low or Intermediate Risk Myelodysplastic Syndromes (MDS) in Japanese Subjects Who Are Not Requiring Red Blood Cell Transfusion

Status(es): Recruiting
Study Date(s): Monday, May 20, 2019 to Sunday, June 16, 2024
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 20 years and older
The study will be conducted in compliance with the International Council for Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 2, multicenter, single-arm study to evaluate the efficacy, safety and Pharmacokinetics (PK) of luspatercept (ACE-536) for the treatment of anemia due to International prognostic scoring system-Revised (IPSS-R) very low, low or intermediate risk Myelodysplastic syndromes (MDS)in Japanese subjects who are not requiring Red blood cell (RBC)...