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.

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...

The National Myelodysplastic Syndromes Natural History Study Spotlight

Status(es): Recruiting
Study Date(s): Friday, April 1, 2016 to Wednesday, September 1, 2021
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
Multi-center study enrolling patients suspected or newly diagnosed with myelodysplastic syndromes (MDS), myelodysplastic syndromes/myeloproliferative neoplasms (MDS/MPN) overlap disorder, or idiopathic cytopenia of undetermined significance (ICUS).  Participants will be followed long term.  Clinical data, blood, and tissue samples will be collected to establish a biorepository to facilitate the study of the natural history of MDS.

Trial of the Combination of Bortezomib and Clofarabine in Adults With Relapsed Solid Tumors Spotlight

Status(es): Recruiting
Study Date(s): Wednesday, August 6, 2014 to Friday, August 31, 2018
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is an open-label Phase I trial. The starting dose of clofarabine will be 1 mg/m2 administered intravenously on days 1 through 5 of a 21-day cycle; bortezomib will be administered at 0.8 mg/m2 subcutaneously on days 1 and 4 of a 21-day cycle. Dose escalation will follow a 3+3 design, with dose limiting toxicities defined during cycle 1. Dose escalation will proceed in cohorts comprised of two separate groups of patients (one group of patients with solid tumor/lymphoma and one group of patients with MDS), with at least 1 from each group, until hematologic DLT or the second grade 2...