Clinical Trials | Page 4 | 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 Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria

Status(es): Recruiting
Study Date(s): Wednesday, August 7, 2019 to Thursday, July 1, 2021
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This is a randomised Phase III, double-blind, multicentre, cross-over study to compare the efficacy, safety, pharmacokinetics, and immunogenicity between SB12 and Soliris® in subjects with PNH.

A Prospective, Multicentre European Registry for Newly Diagnosed Patients With Myelodysplastic Syndromes (EUMDS)

Status(es): Recruiting
Study Date(s): Tuesday, April 1, 2008 to Thursday, December 31, 2020
Disease(s): acute myeloid leukemia (AML), chronic myelomonocytic leukaemia (CMML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Study Objectives: To collect and describe demographics, disease-management, and treatment outcomes of Myelodysplastic Syndromes (MDS) patients who are newly diagnosed and classified according to the WHO criteria. To perform observational studies concerning relevant scientific research questions in MDS using clinical data and biological samples, and to present relevant research outcomes in the fields of diagnosis and prognostication, health related quality of life issues, health economics, and risk stratification for newly developed classes of drugs. To disseminate results of the studies to...

A Randomized Phase II Study of Hyperbaric Oxygen in Improving Engraftment in Umbilical Cord Blood Stem Cell Transplant (HBO-UBC)

Status(es): Recruiting
Study Date(s): Tuesday, November 13, 2018 to Friday, April 30, 2021
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years to 70 years
The UCB transplant is a type of stem cell transplant used to treat cancer of the blood or lymph glands. The UCB transplant has advantages over other types of transplants such as ease of obtaining the umbilical cord blood, absence of donor risks, reduced risks of contagious infections, and the availability for immediate use. The UCB transplant is also associated with a lower incidence of graft versus host disease, or GvHD (in GvHD, the transplanted graft attacks the recipient organs).

A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH (DAHLIA)

Status(es): Recruiting
Study Date(s): Wednesday, April 24, 2019 to Saturday, June 5, 2021
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
This is a randomized, double-blind, active-controlled phase 3 study of ABP 959 in participants with paroxysmal nocturnal hemoglobinuria.

A Study Evaluating the Safety and Pharmacokinetics of Atezolizumab Administered in Combination With Hu5F9-G4 to Patients With Relapsed and/or Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, October 8, 2019 to Friday, November 19, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This Phase Ib study is designed to evaluate the safety and pharmacokinetics of atezolizumab when given in combination with Hu5F9-F4 to patients with relapsed or refractory (R/R) acute myeloid leukemia (AML).

A Study Evaluating Venetoclax in Combination With Azacitidine in Subjects With Treatment-Naïve Higher-Risk Myelodysplastic Syndromes (MDS)

Status(es): Recruiting
Study Date(s): Monday, October 24, 2016 to Wednesday, June 30, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a Phase 1b, open-label, non-randomized, multicenter, dose-finding study evaluating venetoclax in combination with azacitidine in subjects with treatment-naïve higher-risk MDS comprising a dose-escalation portion and a safety expansion portion.

A Study Investigating the Safety, Tolerability and Efficacy of ASP7517 in Subjects With Relapsed/Refractory Acute Myeloid Leukemia (AML) and Relapsed/Refractory Higher Risk Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Thursday, September 19, 2019 to Tuesday, February 28, 2023
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this study is to evaluate the safety and tolerability and to determine the recommended phase 2 dose (RP2D) and/or the maximum tolerated dose (MTD) of ASP7517. This study will also evaluate the clinical response of ASP7517 as well as other measures of anticancer activity of ASP7517.

A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS

Status(es): Recruiting
Study Date(s): Wednesday, October 1, 2014 to Tuesday, June 30, 2020
Disease(s): acute myeloid leukemia (AML), myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.

A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemothe

Status(es): Recruiting
Study Date(s): Monday, August 1, 2016 to Friday, December 31, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a clinical study for adult patients who have recently been diagnosed with acute myeloid leukemia or AML. AML is a type of cancer. It is when bone marrow makes white blood cells that are not normal. These are called leukemia cells. Some patients with AML have a mutation, or change, in the FLT3 gene. This gene helps leukemia cells make a protein called FLT3. This protein causes the leukemia cells to grow faster. For patients with AML who cannot receive standard chemotherapy, azacitidine (also known as Vidaza®) is a current standard of care treatment option in the United States. This...

A Study of ASP2215 (Gilteritinib) Combined With Atezolizumab in Patients With Relapsed or Treatment Refractory FMS-like Tyrosine Kinase (FLT3) Mutated Acute Myeloid Leukemia (AML)

Status(es): Recruiting
Study Date(s): Wednesday, June 19, 2019 to Wednesday, August 31, 2022
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
The purpose of this study is to determine the safety and tolerability of gilteritinib given in combination with atezolizumab in participants with relapsed or treatment refractory FMS-like tyrosine kinase 3 (FLT3) mutated AML and to determine the composite complete remission (CRc) rate for participants who either discontinued the study or completed 2 cycles of gilteritinib given in combination with atezolizumab. This study will also evaluate pharmacokinetics (PK), response to treatment, remission and survival. Adverse events (AEs), clinical laboratory results, vital signs, electrocardiograms...