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
Controlled Study of Rigosertib
Versus Physician’s Choice of
Treatment in MDS Patients
After Failure of an HMA
(INSPIRE) (NCT02562443)
Study Design: Randomized, parallelassignment, open-label safety/efficacy
study
Study Start Date: October 2015
Estimated Study Completion Date: September 2018
Study Status: Currently recruiting participants
Estimated Enrollment: 225
Sponsor: Onconova Therapeutics, Inc.
Patients with myelodysplastic syndromes
(MDS) for whom the hypomethylating
agents (HMA) azacitidine or decitabine
have a poor prognosis have limited treatment options. In a previous study, ONTIME,
the multikinase inhibitor rigosertib did not
improve survival, compared with conventional care, for patients for whom HMAs fail
(median survival = 8.2 months for rigosertib
vs. 5.9 months for supportive care with or
without low-dose cytarabine). However,
patients who were primary-refractory to
HMAs seemed to fare better. The INSPIRE
study has a similar design to the ONTIME
trial but enriches for higher-risk patients
with no or transient responses to HMAs.
A Biomarker-Directed Phase
2 Trial of SY-1425 in Patients
With Acute Myeloid Leukemia
or Myelodysplastic Syndrome
(NCT02807558)
Study Design: Open-label, single-group
assignment, safety/efficacy study
Study Start Date: July 2016
Estimated Study Completion Date: March
2019
Study Status: Currently recruiting participants
Estimated Enrollment: 40
Sponsor: Syros Pharmaceuticals
While retinoids such as tretinoin are the
backbone of treatment of patients with
acute promyelocytic leukemia (APL), a
subset of patients with non-APL acute
myeloid leukemia (AML) or MDS will
respond to treatment with retinoic acid
derivatives. Tamibarotene is a retinoid
32
analogue licensed for treatment of APL
in Japan. In this study, patients who have
a gene expression signature (RARA or
IRF8 genes) that pre-clinical testing has
suggested indicates a higher likelihood of
response to tamibarotene will be eligible
for treatment with this drug.
BLEEDING DISORDERS
Advance your
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ABSTRACTS!
Alice Ma, MD
University of North Carolina School of
Medicine
A Phase 1/2, Dose-Escalation
Safety, Tolerability and
Efficacy Study of BMN 270,
an Adenovirus-Associated
Virus Vector-Mediated Gene
Transfer of Human Factor
VIII in Patients With Severe
Haemophilia A (NCT02576795)
Study Design: Open-label, single-group
assignment safety/efficacy study
Study Start Date: August 2015
Estimated Study Completion Date: June 2021
Study Status: Participant recruitment
suspended
Estimated Enrollment: 12
Sponsor: BioMarin Pharmaceutical, Inc.
Novel gene-based therapies for hemophilia A have lagged behind those for hemophilia B, in part due to the large size of
the factor VIII gene (>8kB, compared with
1.4 kB for factor IX). Recently, the bleeding disorders community was pleasantly
stunned by initial results from the phase
I/II study of an adenovirus-associated virus vector (AAV)-mediated gene therapy
(BMN 270) for hemophilia A sponsored by
BioMarin Pharmaceuticals. These initial
results, released to shareholders, showed
initial promising results for two patients
who had been treated at the highest vector particle dose, achieving initial factor
VIII levels of 57 percent and 60 percent.
In March 2016, BMN 270 was granted
orphan drug designation by the U.S. Food
and Drug Administration for hemophilia A.
Recruitment has been temporarily
suspended while the study sponsor and
E.U. regulators discuss the trial prior
to starting BMN 270 in the last three
patients enrolled in the trial. Almost all
pharmaceutical companies performing
factor IX gene therapy trials are turning
their sights on factor VIII as well, though
these clinical trials are not yet open for
recruitment. It is anticipated that the
trial will begin again. ●
Present your
innovative ideas
alongside top experts
in the field
Build relationships
with practice
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Abstracts may be resubmitted to the 2016 ASH Annual Meeting.
2016 ASH
Meeting on
Hematologic
Malignancies
®
SEPTEMBER 16-17, 2016
Learn more and submit an abstract at
hematology.org/malignancies.
ASH Clinical News
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