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 Phase III, International, Randomized, Controlled Study of Rigosertib versus Physician’s Choice of Treatment in Patients with Myelodysplastic Syndrome after Failure of a Hypomethylating Agent Spotlight

Status(es): Recruiting
Study Date(s): Thursday, October 1, 2015 to Sunday, September 30, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 - 81 years
Patients with higher-risk MDS failing HMA treatment (targeted patient population)  have no approved medical therapy and a short life expectancy (approximately 4-6 months; Bello 2009, Jabbour 2008, Jabbour 2010, Prebet 2011). Once treatment with an HMA has failed prognosis is dismal and thus there is a great unmet medical need to identify  novel therapies for these patients. We propose to enroll patients with MDS who have excess blasts (5% to 30% BM blasts), and have progressed during, failed to respond to, are intolerant of, or relapsed after AZA or DAC treatment. Patients’ MDS must be...

A Phase 1, Multicenter, Open-Label, Dose-Escalation and Expansion, Safety, Pharmacokinetic, Pharmacodynamic, and Clinical Activity Study of Orally Administered AG-120 in Subjects with Advanced Hematologic Malignancies with an IDH1 Mutation Spotlight

Status(es): Recruiting
Study Date(s): Monday, March 17, 2014 to Friday, June 30, 2017
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, pharmacokinetics, pharmacodynamics and clinical activity of AG-120 in advanced hematologic malignancies that harbor an IDH1 mutation.

A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects with PNH Spotlight

Status(es): Recruiting
Study Date(s): Monday, November 24, 2014
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of the study is to assess the safety, tolerability and pharmacokinetics of APL-2 in subjects with paroxysmal nocturnal hemoglobinuria (PNH) who are still anemic after treatment with eculizumab (Soliris®).

AK579 (COBALT) Spotlight

Status(es): Recruiting
Study Date(s): Monday, August 1, 2016 to Friday, June 30, 2017
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
Coversin Global Study:  An Open-Label, Safety and Efficacy Trial in PNH Patients

CASCADE (SGN33A-005) Spotlight

Status(es): Recruiting
Study Date(s): Wednesday, May 25, 2016 to Saturday, September 25, 2021
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
The purpose of this study in AML patients is to test whether vadastuximab talirine (SGN-CD33A; 33A) combined with either azacitidine or decitabine extends overall survival longer than placebo combined with either azacitidine or decitabine.

Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms Spotlight

Status(es): Recruiting
Study Date(s): Monday, February 8, 2016 to Sunday, January 1, 2017
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
Coversin in paroxysmal nocturnal haemoglobinuria (PNH) in patients with resistance to eculizumab due to complement C5 polymorphisms

MEDALIST Trial Spotlight

Status(es): Recruiting
Study Date(s): Tuesday, December 1, 2015 to Tuesday, June 25, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 and older
The study will be conducted in compliance with the International Council on Harmonisation (ICH) of Technical Requirements for Registration of Pharmaceuticals for Human Use/Good Clinical Practice (GCP) and applicable regulatory requirements. This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in subjects with anemia due to International Prognostic Scoring System-Revised (IPSS-R) very low, low, or intermediate Myelodysplastic syndrome (MDS) with ring sideroblasts (≥ 15%) who require Red...

Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients Who Have an Inadequate Response to Eculizumab Spotlight

Status(es): Not yet recruiting
Study Date(s): Saturday, April 1, 2017 to Sunday, April 1, 2018
Disease(s): paroxysmal nocturnal hemoglobinuria (PNH)
Age Group: 18 years and older
The purpose of the study is to evaluate the safety and efficacy of RA101495 in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have an inadequate response to eculizumab. Patients will be treated with RA101495 for 12 weeks. Note: Patients on the study will receive RA101495 at the dose of 0.3mg/kg subcutaneously (SC) at Day 1 (loading dose) followed by a starting maintenance dose of 0.1 mg/kg daily SC

REVERSE 1 INCB 18424-271 Spotlight

Status(es): Recruiting
Study Date(s): Tuesday, November 15, 2016 to Friday, September 29, 2017
Disease(s): graft versus host disease (GVHD)
Age Group: 12 and older
A Single-Cohort, Phase 2 Study of Ruxolitinib in Combination With Corticosteroids for the Treatment of Steroid-Refractory Acute Graft-Versus-Host Disease - The purpose of this study is to assess the efficacy of ruxolitinib in combination with corticosteroids in subjects with Grades II to IV steroid-refractory acute graft-versus-host disease (GVHD).

Selinexor (KPT-330) in Older Patients With Relapsed/Refractory AML (SOPRA) Spotlight

Status(es): Recruiting
Study Date(s): Saturday, March 1, 2014 to Thursday, June 1, 2017
Disease(s): acute myeloid leukemia (AML)
Age Group: 18 years and older
This is a randomized, multicenter, open-label, Phase 2 study of the oral SINE™ compound selinexor in patients 60 years of age or older with relapsed or refractory AML who are ineligible for standard intensive chemotherapy and/or transplantation. In the Selinexor in Older Patient with Relapsed/Refractory AML (SOPRA) study, Karyopharm is evaluating approximately 170 patients who have AML that has relapsed after, or was refractory to, first line therapy. Patients are randomized in a 2:1 fashion to selinexor provided orally twice weekly in a dose of 60mg plus best supportive care (BSC) versus...