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 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): 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): acute myeloid leukemia (AML)
Age Group: 18 years and older
Coversin Global Study:  An Open-Label, Safety and Efficacy Trial in PNH Patients

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

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...

Prior allogeneic transplant Karnofsky Performance Score < 70% Active central nervous system (CNS) involvement by malignant cells Patients with uncontrolled bacterial, viral or fungal infections (currently taking medication and with progre

Status(es): Recruiting
Study Date(s): Friday, April 1, 2011 to Sunday, October 1, 2017
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 3 months to 30 years
This randomized phase III trial studies caspofungin acetate to see how it works compared to fluconazole in preventing invasive fungal infections in patients with acute myeloid leukemia who are undergoing chemotherapy. Caspofungin acetate or fluconazole may help prevent fungal infections caused by chemotherapy. It is not yet known whether fluconazole is more effective than caspofungin acetate in preventing fungal infections in patients with acute myeloid leukemia who are undergoing chemotherapy.

3'-Deoxy-3'-[18F] Fluorothymidine PET Imaging in Patients With Cancer

Status(es): Recruiting
Study Date(s): Tuesday, September 1, 2009 to Saturday, September 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years to 120 years
RATIONALE: Diagnostic procedures, such as 3'-deoxy-3'-[18F] fluorothymidine (FLT) PET imaging, may help find and diagnose cancer. It may also help doctors predict a patient's response to treatment and help plan the best treatment. PURPOSE: This phase I trial is studying FLT PET imaging in patients with cancer.

8-Chloro-Adenosine in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia

Status(es): Recruiting
Study Date(s): Tuesday, September 1, 2015 to Saturday, September 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This phase I/II trial studies the side effects and best dose of 8-chloro-adenosine and how well it works in treating patients with acute myeloid leukemia that has returned after a period of improvement (relapsed) or has not responded to previous treatment (refractory). Drugs used in chemotherapy, such as 8-chloro-adenosine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading.

A Biomarker-Directed Phase 2 Trial of SY-1425 in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome

Status(es): Recruiting
Study Date(s): Monday, August 1, 2016 to Friday, March 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
The purpose of this study is to determine the activity of SY-1425 in patients with relapsed/refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) who are positive for a RARA super-enhancer associated biomarker.

A Combination Study of PF-04449913 and Azacitidine In 1st Line MDS, AML and CMML Patients

Status(es): Recruiting
Study Date(s): Wednesday, April 1, 2015 to Friday, June 1, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This multi center randomized (1:1), double blind, placebo controlled Phase 1b/2 study is designed to compare the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of PF 04449913 or placebo when combined with azacitidine in patients with previously untreated Intermediate 2 or High RiskMyelodysplastic Syndrome (MDS), Acute Myeloid Leukemia (AML) with 20-30% blasts and multi lineage dysplasia, and Chronic Myelomonocytic Leukemia (CMML). This clinical study includes two components: (a) a Phase 1b safety lead in and (b) a randomized Phase 2.