Trial Roundup
ASH Clinical News’ Associate Editors select
clinical trials to keep an eye on.
LEUKEMIA
BLEEDING DISORDERS
estimated study completion date:
David Steensma, MD
Dana-Farber Cancer Institute
Alice Ma, MD
University of North Carolina School of Medicine
study status:
A Phase I, Multicenter, Open-label
Study of Oral ABL001 in Patients With
Chronic Myelogenous Leukemia (CML)
or Philadelphia Chromosome-positive
Acute Lymphoblastic Leukemia (Ph+
ALL) (NCT02081378)
Pharmacologic prevention and treatment of sickle
cell crises has relied on a single agent – hydroxyurea
– for many years. However, a recent spate of promising new agents are now being evaluated in clinical
trials. These new therapies have a variety of targets:
blocking adhesion receptors, increasing fetal hemoglobin, altering the oxygen affinity of sickle cell
hemoglobin, blocking ischemia/reperfusion injury,
and altering the arginine metabolome. In the February issue, we discussed trials targeting selectins
in sickle cell disease (SCD), and in the March issue
we focused on HDAC inhibitors in SCD. I’ll close the
sickle cell roundup with drugs that target integrins
and other adhesive molecules in SCD.
study design:
Non-randomized, open-label, singlegroup assignment safety study
study start date: April 2014
estimated study completion date: February 2017
study status: Currently recruiting participants
estimated enrollment: 60
sponsor: Novartis Pharmaceuticals
Patients with chronic myeloid leukemia (CML) and Ph+
acute lymphoblastic leukemia (ALL) often develop
resistance to tyrosine kinase inhibitor therapy (TKI) via
ABL kinase point mutations, and even patients who
achieve deep molecular remissions frequently relapse
after TKI discontinuation. All currently available TKIs
for CML and Ph+ ALL bind to the ATP binding pocket
of the ABL1 kinase, and mutations that alter the
conformation of this pocket induce remission. The
small molecule ABL001, in contrast, was developed
to target the myristoyl pocket of the ABL1 kinase and
inhibits proliferation of BCR-ABL positive cells with
clinically observed TKI resistance mutations. This
study is designed to assess the safety, tolerability, and
clinical activity of ABL001 in patients with CML or Ph+
ALL who have either developed resistance to at least
TKIs or have the T315I ABL mutation and are resistant
to at least one TKI.
A Dose Escalation and Cohort
Expansion Study of TEN-010 in
Patients With Acute Myeloid Leukemia
and Myelodysplastic Syndromes
(NCT02308761)
study design:
Open-label, single-group assignment,
safety study
study start date:
October 2014
estimated study completion date: April
2017
Currently recruiting participants
estimated enrollment: 68
sponsor: Tensha Therapeutics
study status:
Evaluation of Purified Poloxamer 188
in Vaso-Occlusive Crisis of Sickle Cell
Disease (EPIC) (NCT01737814)
study design:
Randomized, double-blind, parallelassignment efficacy study
study start date: May 2013
estimated study completion date: December 2015
study status: Currently recruiting participants
estimated enrollment: 388
sponsor: Mast Therapeutics, Inc.
MST-188, purified polaxamer 188, is a synthetic polymer
of polyoxyethylene (POE) and polyoxypropylene (POP)
thought to have beneficial effects on blood flow, as
well as having anti-inflammatory and antithrombotic
effects. An initial tria