CLINICAL NEWS
Trial Roundup
LEUKEMIA
David Steensma, MD
Dana-Farber Cancer Institute
A Phase 2, Open Label, Ascending Dose
Study of ACE-536 for the Treatment
of Anemia in Patients With Low or
Intermediate-1 Risk Myelodysplastic
Syndromes (NCT01749514)
reperfusion injury, and altering the arginine metabolome. Last month, I focused on trials targeting selectins in sickle cell disease (SCD). This month’s roundup
focuses on histone deacetylase (HDAC) inhibitors.
HDAC inhibitors were first tried in treatment of malignancies but were also noted to increase levels of fetal
hemoglobin (HbF). Additionally, HDAC inhibitors have
been shown to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating
effects of SCD.
study design:
Open-label, single-group assignment
safety/efficacy study
study start date: January 2013
estimated study completion date: April 2015
study status: Currently recruiting participants
estimated enrollment: 60
sponsor: Acceleron Pharma, Inc.
Patients with myelodysplastic syndromes (MDS)–
associated anemia who have not responded to
erythropoiesis-stimulating agents (ESAs), such as
darbepoetin and epoetin, have limited treatment options. Luspatercept, or ACE-536, is an activin receptor
IIB ligand trap, which decreases levels of ligands in the
transforming growth factor-β superfamily, including
GDF11. GDF11 inhibits erythropoiesis at a later stage of
red cell development than that altered by ESAs. Early
data presented at the 2014 ASH Annual Meeting in
December suggest that ACE-536 improves anemia in
some patients with MDS who have not responded to
or are unlikely to respond to ESAs.
APG101 in Transfusion-Dependent
Patients With Low or Intermediate
Risk Myelodysplastic Syndrome
Efficacy of Vorinostat to Induce Fetal
Hemoglobin in Sickle Cell Disease
(NCT01000155)
study design:
Open-label, single-group assignment,
safety/efficacy study
study start date: October 2009
estimated study completion date: October 2014
study status: Currently recruiting participants
estimated enrollment: 15
sponsor: Dana-Farber Cancer In 7F