CLINICAL NEWS
Trial Roundup
ASH Clinical News’ Associate Editors select clinical trials to keep an eye on.
LEUKEMIA
BLEEDING DISORDERS
LYMPHOMA & MYELOMA
David Steensma, MD
Dana-Farber Cancer Institute
Alice Ma, MD
University of North Carolina School of
Medicine
Keith Stewart, MBChB, MBA
Mayo Clinic, Arizona
Ruxolitinib and Pracinostat
Combination Therapy for Patients
With Myelofibrosis (NCT02267278)
study design:
Open-label, single-group assignment safety/efficacy study
study start date: January 2015
estimated study completion date: January 2018
study status: Currently recruiting participants
estimated enrollment: 25
sponsor: MD Anderson Cancer Center, MEI
Pharma
Ruxolitinib is an established palliative
therapy for patients with myelofibrosis and
offers the possibility of improvement in
splenomegaly and constitutional symptoms. In vitro analysis has shown that
there is synergy between JAK2 inhibitors
(like ruxolitinib) and deacetylase inhibitors
(like pracinostat). Pracinostat has shown
activity in myelodysplastic syndrome when
used in combination with hypomethylating
agents. This trial is an example of one of
several ongoing trials evaluating combination therapy regimens with JAK2 inhibitors
in myelofibrosis.
Phase 2 Dose-Ranging Trial
of Ponatinib in Patients With
Refractory, Chronic-Phase Chronic
Myeloid Leukemia
Study to Assess Safety and
Impact of SelG1 With or Without
Hydroxyurea Therapy in Sickle Cell
Disease Patients With Pain Crises
(SUSTAIN) (NCT01895361)
study design:
Randomized, double-blind parallel
assignment study
study start date: July 2013
estimated study completion date: August 2015
study status: Currently recruiting participants
estimated enrollment: 174
sponsor: Selexys Pharmaceuticals Corporation
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 is now
in clinical trials. Therapies are now aimed at
blocking adhesion receptors, increasing fetal
hemoglobin, altering the oxygen affinity of
hemoglobin S, blocking ischemia/reperfusion
injury, and altering the arginine metabolome.
The SUSTAIN trial evaluates SelG1, an inhibitor
of p-selectin; in preclinical studies, inhibition
of p-selectin prevented vaso-occlusion by
blocking cell-cell interactions. Based on the
safety demonstrated by SelG1 in a phase 1
trial in normal subjects, this phase 2 trial is
underway to determine if the drug with or
without hydroxyurea impacts the incidence
and severity of painful vaso-occlusive crises.
study design:
Multicenter, dose-finding study
Mid-2015
study status: Not yet recruiting participants
estimated enrollment: 450
sponsor: Ariad Pharmaceuticals
study start date:
Ponatinib is approved for chronic myeloid
leukemia (CML) or Ph+ acute lymphocytic
leukemia (ALL) with either T315I mutation,
or patients for whom all other tyrosine kinase inhibitor (TKI) drugs have failed. When
the drug was originally approved by the
FDA in 2013, it was approved at 45 mg/
day, which may be too high for many patients. The drug was briefly taken off the
market in late 2013 in the United States due
to a higher-than-anticipated rate of cardiovascular events, including vascular occlusion.
Now when we start patients on ponatinib
we usually do so at a dose of 15 or 30 mg/
day. This study will enroll approximately 450
patients at three different starting doses
of ponatinib in order to understand whether
lower doses might be safer or might differ in
effectiveness from higher doses.
ASHClinicalNews.org
Efficacy and Safety of Rivipansel
(GMI-1070) in the Treatment
of Vaso-Occlusive Crisis in
Hospitalized Subjects With Sickle
Cell Disease (NCT02187003)
study design:
Randomized, double-blind, parallel assignment safety/efficacy study
study start date: June 2015
estimated study completion date: July 2018
study status: Not yet open for recruitment
estimated enrollment: 350
sponsor: Pfizer
Rivipansel is a synthetic glycomimetic molecule,
which was rationally designed as a pan-selectin
inhibitor that will inhibit all three selectin types.
A phase 2 randomized, double-blind study in
hospitalized patients with sickle cell disease experiencing veno-occlusive crisis (VOC) found that
rivipansel reduced the time to reach resolution
of VOC, length of hospital stay, and use of opioid
analgesics for pain management. This follow-up
phase 3, multicenter, randomized, double-blind,
placebo-controlled clinical study will evaluate the
safety and efficacy of rivipansel in patients experiencing a pain crisis necessitating hospitalization.
Phase III Study of Lenalidomide and
Dexamethasone With or Without
Elotuzumab to Treat Relapsed
or Refractory Multiple Myeloma
(ELOQUENT - 2) (NCT01239797)
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