Clinical Trials | Page 2 | 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.

A Phase I/II Study of OPN-305 in Second-line Lower Risk Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Thursday, January 1, 2015 to Sunday, May 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The dose-confirming part of this study, comprising at least 10 patients is designed as a single center, prospective, single arm, open label in patients who have failed or are unresponsive to an Erythropoiesis Stimulating Agent (ESA) and/or Azacitidine (AZA) followed by a dose expansion part with an additional 30 patients; the objective of the whole study being to assess the safety, efficacy, pharmacokinetics and pharmacodynamics of intravenously infused multiple doses of OPN-305 in low and intermediate-1 riskmyelodysplastic syndrome (Second line Lower risk MDS).

A Phase Ib/IIb, Open-label, Multi-center, Study of Oral Panobinostat Administered With 5-Azacitidine (in Adult Patients With Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Acute Myeloid Leukemia (AML).

Status(es): Recruiting
Study Date(s): Wednesday, December 1, 2010 to Thursday, December 1, 2016
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this randomized, two-arm, open-label expansion phase study is to collect preliminary efficacy data of panobinostat at the recommended phase II dose (RPIID) level in combination with azacytidine (5-Aza) versus an active control arm 5-Aza alone. This randomized phase II part allows also collecting safety data of panobinostat in combination with 5-Aza in comparison to single-agent 5-aza.

A Phase II Study of 5-Azacitidine and Sargramostim as Maintenance Treatment After Definitive Therapy for Poor-risk AML or MDS

Status(es): Recruiting
Study Date(s): Saturday, June 1, 2013 to Sunday, October 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 6 months to 100 years
We propose a phase II study to determine the impact of maintenance therapy with 5-azacytidine and GM-CSF in patients with poor-risk AML or MDS, who are in remission after definitive treatment with either stem cell transplant or cytarabine-based consolidation chemotherapy. In order to precede relapse and to avoid lead time bias, treatment would need to commence within 185 days of definitive therapy. Furthermore, approximately 50% of relapses occur within the first year and up to 80% within two years after SCT, therefore we would limit the duration of maintenance therapy to one year, followed...

A Phase II Trial of CD24Fc for Prevention of Acute Graft-versus-Host Disease Following Myeloablative Allogeneic Hematopoietic Stem Cell Transplant

Status(es): Recruiting
Study Date(s): Monday, February 1, 2016 to Monday, June 1, 2020
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a multicenter prospective randomized phase IIa/IIb clinical trial designed to determine the MTD (Maximum Tolerated Dose) of CD24Fc for acute GVHD (Graft Versus Host Disease) prophylaxis.

A Pilot Study of a Thrombopoietin-Receptor Agonist, Eltrombopag, in Patients With Low to Int-2 Risk Myelodysplastic Syndrome (MDS)

Status(es): Recruiting
Study Date(s): Wednesday, July 1, 2009 to Saturday, December 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Background: Myelodysplastic syndromes (MDS) are bone marrow disorders characterized by anemia, neutropenia, and thrombocytopenia (low red blood cell, white blood cell, and platelet counts). Patients with MDS are at risk for symptomatic anemia, infection, and bleeding, as well as a risk of progression to acute leukemia. Standard treatments for MDS have significant relapse rates. MDS patients with thrombocytopenia who fail standard therapies require regular, expensive, and inconvenient platelet transfusions, and are at risk for further serious bleeding complications. Eltrombopag is a drug...

A Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)

Status(es): Recruiting
Study Date(s): Friday, May 20, 2011 to Thursday, September 1, 2016
Disease(s): aplastic anemia
Age Group: 2 years and older
Leucine will be provided to participants in the form of a capsule and will be taken three times daily. Blood hemoglobin levels will be monitored every 3-4 weeks for 9 months. The entire study will last 12-15 months in length. Subjects must be two years of age or older and on transfusion for more than six months prior to enrollment.

A Safety and Pharmacology Study of Atezolizumab (MPDL3280A, Anti-PD-L1 Antibody) Administered Alone or in Combination With Azacitidine in Patients With Myelodysplastic Syndromes

Status(es): Recruiting
Study Date(s): Tuesday, September 1, 2015 to Friday, March 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a non-randomized, open-label, Phase 1 study of Atezolizumab (MPDL3280A, Anti-PD-L1 antibody) monoclonal antibody [mAb] in intermediate/high/very high-risk myelodysplastic syndromes (MDS) patients, as evaluated by the International Prognostic Scoring System-Revised (IPSS-R). Eligible participants will either have never received treatment with hypomethylating agents(s) (HMAs) or have relapsed or are refractory to prior HMA therapy. The primary objectives of this study are to determine the safety and feasibility of Atezolizumab therapy in these patient populations, including treatment...

A Sequential Two-Stage Dose Escalation Study to Evaluate the Safety and Efficacy of Ruxolitinib

Status(es): Recruiting
Study Date(s): Friday, February 1, 2013 to Friday, September 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years or older
The purpose of this study is to find out if treating Chronic Myelomonocytic Leukemia (CMML) with a study drug [ruxolitinib] can improve outcomes of patients with CMML. The first step of the study is to learn the dose of ruxolitinib that is tolerable (bearable). It has already been studied in a number of patients with different bone marrow diseases and is approved for the treatment of a disease called Myelofibrosis; however, it is not approved for treatment of CMML. It is given orally (by mouth). Most people tolerate it well but the tolerability has not been determined in patients with CMML....

A Study of BBI608 in Adult Patients With Advanced, Refractory Hematologic Malignancies

Status(es): Recruiting
Study Date(s): Friday, May 1, 2015 to Monday, April 2, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a multicenter, open label, Phase 1 dose-escalation study of BBI608 administered to patients with relapsed, refractory hematologic malignancies, including multiple myeloma, lymphoma, and others.

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.