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...

Study of Vadastuximab Talirine (SGN-CD33A; 33A) in Combination With Azacitidine in Patients With Previously Untreated Higher Risk MDS Spotlight

Status(es): Suspended
Study Date(s): Monday, February 1, 2016 to Saturday, June 1, 2019
Disease(s): myelodysplastic syndromes (MDS)
Age Group: 18 years and older
This is a phase 1/2 study to evaluate the combination of vadastuximab talirine (SGN-CD33A) and azacitidine in subjects with previously untreated International Prognostic Scoring System (IPSS) Intermediate-2 or high risk myelodysplastic syndrome (MDS).  Primary objectives include determining the recommended dose of vadastuximab talirine (SGN-CD33A) in combination with azacitidine (in the open-label Phase 1 portion), and to compare the overall response rate between treatment arms (in the randomized, double-blinded, placebo-controlled Phase 2 portion).

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.