CLINICAL NEWS
Trial Roundup
ASH Clinical News’ Associate Editors select
clinical trials to keep an eye on.
LEUKEMIA
David Steensma, MD
Dana-Farber Cancer Institute
Oral Pacritinib Versus Best Available
Therapy to Treat Myelofibrosis With
Thrombocytopenia (NCT02055781)
study design:
gemtuzumab, if the efficacy of this new agent
outweighs its toxicity and, if inotuzumab is approved,
this agent will provide additional options for patients
with relapsed/refractory ALL. These new agents are
likely to be tried soon in the upfront setting – adding
to the current combination chemotherapy regimens.
BLEEDING DISORDERS
Randomized, open-label, parallel assignment safety/efficacy study
study start date: December 2013
estimated study completion date: April 2018
study status: Currently recruiting participants
estimated enrollment: 300
sponsor: CTI BioPharma
Alice Ma, MD
University of North Carolina School of Medicine
Currently, ruxolitinib is the only JAK2 inhibitor
approved for myelofibrosis. While this agent is
effective at reducing splenomegaly and improving
constitutional symptoms in many patients,
treatment-emergent thrombocytopenia can be
problematic, especially for patients with pretreatment thrombocytopenia from the underlying
disease. Pacritinib (formerly known as SB1518)
appeared to cause less thrombocytopenia than
ruxolitinib in earlier phase trials. The drug is now
being compared in a randomized fashion with “best
available therapy” in patients with myelofibrosis and
splenomegaly. Best available therapy includes any
available myelofibrosis therapy, including ruxolitinib
or hydroxyurea, glucocorticoids, erythropoietic
agents, immunomodulatory agents, mercaptopurine,
danazol, interferons, cytarabine, melphalan, or other
agents, and may also include no treatment and
symptom-directed treatment without myelofibrosisspecific treatment.
study design:
A Study of Inotuzumab Ozogamicin Versus
Investigator’s Choice of Chemotherapy
in Patients With Relapsed or Refractory
Acute Lymphoblastic Leukemia
The use of the new oral anticoagulant agents has
been hampered by lack of safe, effective specific
reversal agents. Andexanet alpha, a novel, first-inclass molecule, is being tested for its ability to reverse the anticoagulant effects of both rivaroxaban
and apixaban, two direct factor Xa inhibitors. Andexanet is a modified factor Xa molecule that lacks
a GLA-domain – making it unable to bind to membranes – and is also catalytically inactive. Andexanet
competes with native factor Xa for binding to rivaroxaban and apixaban (and presumably other factor
Xa inhibitors), serving as a decoy and limiting drug
availability. Andexanet alfa has been designated as a
“breakthrough therapy” by the FDA, and these phase
3 studies are continuing to recruit patients.
(NCT 01564784)
study design:
Randomized, open-label, parallel assignment safety/efficacy study
study start date: August 2012
estimated study completion date: March 2017
study status: Currently recruiting participants
estimated enrollment: 325
sponsor: Pfizer
The FDA recently granted accelerated approval for
relapsed/refractory B-cell acute lymphocytic leukemia
(ALL) blinotumumab, a bispecific CD19-CD3 antibody
– the first new class of drug for B-cell ALL to achieve
regulatory approval in many years. Inotuzumab, a
CD22 antibody conjugated to the same toxin used
in the formerly approved AML drug gemtuzumab
ozogamicin, showed dramatic activity in earlyphase trials of relapsed/refractory B-ALL expressing
CD22. Currently, inotuzumab is being compared
in a randomized fashion to three commonly used
regimens (FLAG, HAM, and high-dose cytarabine).
While the incidence of