Clinical Trials | Page 4 | 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:

A Study of CC-90002 in Subjects With Acute Myeloid Leukemia (AML) and High-risk Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Tuesday, March 1, 2016 to Monday, September 2, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Study CC-90002-AML-001 is an open-label, Phase 1 dose escalation (Part A) and expansion (Part B), clinical study of CC-90002, administered by intravenous (IV) infusion, in subjects with relapsed and/or primary refractory AML and high-risk MDS. The study will explore escalating doses of CC-90002 using a 3 + 3 dose escalation design in Part A, followed by dose expansion in Part B. The primary objective is to determine the safety and tolerability of CC-90002 and also to define the non-tolerated dose (NTD), the maximum tolerated dose (MTD) and/or the recommended Phase 2 dose (RP2D) of CC-90002.

A Study of DSP-7888 Dosing Emulsion in Adult Patients With Advanced Malignancies

Status(es): Recruiting
Study Date(s): Sunday, November 1, 2015 to Wednesday, November 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a multicenter, open label, Phase 1 dose-escalation study of DSP-7888 Dosing Emulsion administered to adult patients with advanced malignancies. Patients will be administered escalating doses of DSP-7888 Dosing Emulsion intradermally in accordance with the following regimen: once weekly for four weeks during the Induction Phase, once every 7 to 14 days for 6 weeks during the Consolidation Phase, and once every 14 to 28 days until a discontinuation criterion is met during the Maintenance Phase.

A Study of E6201 for the Treatment of Advanced Hematologic Malignancies With FLT3 Mutation (AML)

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2015 to Sunday, October 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years or older
This is a Phase 1/2a dose-escalation study of E6201, a dual MEK1 and FLT3 inhibitor, in subjects with advanced hematologic malignancies with documented FLT3 mutation. The Phase1 portion of the study will be a safety run-in to establish a recommended Phase 2 dose (RP2D). The Ph. 2a portion of the study will evaluate two specific patients groups: Cohort 1 will enroll up to 26 patients with relapsed or refractory AML and confirmed FLT3 mutation without prior exposure to a FLT3 inhibitor while Cohort 2 will enroll up to 12 patients with relapsed or refractory AML and confirmed FLT3 mutation with...

A Study of Eltrombopag or Placebo in Combination With Azacitidine in Subjects With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-risk Myelodysplastic Syndromes (MDS)

Status(es): Recruiting
Study Date(s): Sunday, June 1, 2014 to Friday, December 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Eltrombopag olamine (SB-497115-GR) is an orally bioavailable, small molecule thrombopoietin receptor agonist that may be beneficial in medical disorders associated with thrombocytopenia. Eltrombopag has been shown to increase platelet counts in patients with thrombocytopenia from various etiologies (Idiopathic thrombocytopenic purpura [ITP], liver disease, aplastic anemia and chemotherapy induced thrombocytopenia). Approximately 350 subjects will be randomized in a 1:1 ratio (175 into the eltrombopag arm and 175 into the placebo arm). Approximately 55 subjects will be enrolled into the...

A Study of Luspatercept (ACE-536) to Treat Anemia Due to Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MEDALIST)

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Tuesday, January 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
A PHASE 3, DOUBLE-BLIND, RANDOMIZED STUDY TO COMPARE THE EFFICACY AND SAFETY OF LUSPATERCEPT (ACE-536) VERSUS PLACEBO FOR THE TREATMENT OF ANEMIA DUE TO IPSS-R VERY LOW, LOW, OR INTERMEDIATE RISK MYELODYSPLASTIC SYNDROMES IN SUBJECTS WITH RING SIDEROBLASTS WHO REQUIRE RED BLOOD CELL TRANFUSIONS

A Study of PLX51107 in Advanced Malignancies

Status(es): Recruiting
Study Date(s): Tuesday, March 1, 2016 to Monday, January 1, 2018
Disease(s):
Age Group: 18 years and older
The purpose of this research study is to evaluate safety, pharmacokinetics, pharmacodynamics, and preliminary efficacy of the investigational drug PLX51107 in subjects with advanced solid tumors (including lymphoma), acute myeloid leukemia (AML), or high-risk myelodysplastic syndrome (MDS).

A Study To Evaluate PF-04449913 With Chemotherapy In Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Friday, June 1, 2012 to Thursday, September 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a study to evaluate PF-04449913 (an inhibitor of the Hedgehog pathway) in Acute Myeloid Leukemia and high-risk Myelodysplastic Syndrome in combination with standard agents used to treat these diseases.

A Trial of Pembrolizumab (MK-3475) in Participants With Blood Cancers (MK-3475-013)(KEYNOTE-013)

Status(es): Recruiting
Study Date(s): Friday, November 1, 2013 to Sunday, January 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this trial is to evaluate the safety, tolerability, and efficacy of pembrolizumab (MK-3475, KEYTRUDA®) in hematologic malignancies. The primary study hypotheses are that treatment with pembrolizumab will result in a clinically meaningful improvement in Objective Response Rate (ORR) or Complete Remission Rate (CRR)

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...

A Two-Step Approach to Reduced Intensity Bone Marrow Transplant for Patients With Hematological Malignancies

Status(es): Recruiting
Study Date(s): Monday, June 27, 2011 to Monday, August 1, 2016
Disease(s): aplastic anemia
Age Group: 18 years and older
PRIMARY OBJECTIVES: I. To compare the overall survival (OS) rate at 2 years post treatment using the Jefferson 2 step reduced intensity conditioning (RIC) approach in patients with haploidentical family donors with hematological malignancies in morphological or radiographic remission or with chemosensitive, indolent diseases to historical OS rates in similar populations after RIC matched donor HSCT as reported in the literature. SECONDARY OBJECTIVES: I. To compare the treatment-related mortality (TRM) rate at 2 years for patients treated on this study to the historical TRM rates of patients...