CLINICAL NEWS
Trial Roundup
ASH Clinical News’ Associate Editors select
clinical trials to keep an eye on.
LEUKEMIA
BLEEDING DISORDERS
David Steensma, MD
Dana-Farber Cancer Institute
Alice Ma, MD
University of North Carolina School of Medicine
Phase 1 Trial to Evaluate the Safety,
Pharmacokinetics and Pharmacodynamics
of Splicing Modulator H3B-8800 for
Subjects With Myelodysplastic Syndromes,
Acute Myeloid Leukemia, and Chronic
Myelomonocytic Leukemia (NCT02841540)
Minimize Menorrhagia in Women With
Type 1 Von Willebrand Disease (VWDMin)
Study Design: Non-randomized, parallel-assignment,
open-label safety/efficacy study
Study Start Date: August 2016
Estimated Study Completion D ate: March 2019
Study Status: Currently recruiting participants
Estimated Enrollment: 110
Sponsor: H3 Biomedicine Inc.
The investigational agent H3B-8800 is an oral and
selective small molecule modulator of splicing factor 3b
subunit 1. The agent demonstrated antitumor activity in
several pre-clinical xenograft models carrying spliceosome mutations. This study is composed of a dose-finding phase (cohorts A and B) and a four-arm expansion
phase. The study will determine the maximum-tolerated
dose and recommended phase II dose of H3B-8800 in
patients with myelodysplastic syndromes, acute myeloid
leukemia, or chronic myelomonocytic leukemia; and it
will assess the safety and tolerability of H3B-8800 as a
single agent in these patients.
Study to Determine Safety,
Pharmacokinetics and Efficacy of GMI-1271
in Combination With Chemotherapy in
AML (NCT02306291)
Study Design: Non-randomized, single-group assignment, open-label safety/efficacy study
Study Start Date: March 2015
Estimated Study Completion Date: December 2018
Study Status: Currently recruiting participants
Estimated Enrollment: 102
Sponsor: GlycoMimetics Incorporated
This study will evaluate the safety and efficacy of GMI1271, in combination with a standard 7+3 regimen, for
the treatment of patients with acute myeloid leukemia
(AML). GMI-1271 blocks E-selectin (an adhesion molecule
on cells in the bone marrow) from binding with AML cells
to disrupt mechanisms of leukemic cell resistance within
the bone marrow microenvironment. Earlier this year,
GMI-1271 was granted fast-track designation by the U.S.
Food and Drug Administration for this indication.
(NCT02606045)
Study Design: Randomized, crossover assignment,
open-label efficacy study
Study Start Date: July 2016
Estimated Study Completion Date: June 2021
Study Status: Not yet recruiting participants
Estimated Enrollment: 60
Sponsor: University of Pittsburgh
This is an outpatient, 24-week, phase III prospective, randomized, crossover trial comparing recombinant von Willebrand
factor (vWF) and tranexamic acid to minimize menorrhagia in
women with type 1 vWD. The trial is not yet open for participant recruitment, but results should answer the question of
comparative efficacy of vWF replacement therapy versus an
antifibrinolytic agent in treating menorrhagia.
Investigating a von Willebrand Factor
Functional Screening Assay for Assigning
the Phenotypic Variants of von Willebrand
Disease (vWF-phV) (NCT02466789)
Study Design: Prospective, observational study
Study Start Date: July 2015
Estimated Study Completion Date: December 2019
Study Status: Currently recruiting participants
Estimated Enrollment: 100
Sponsor: Blood Center of Wisconsin
This study investigates a new blood screening test for the
diagnosis of vWD, and will compare its accuracy and the rapidity with which it makes a diagnosis with other available
methods. The test being studied is a novel ELISA-based
vWF functional screening assay, and, unlike current tests,
it would be available at local hospital labs as opposed to requiring samples to be sent to larger, more specialized labs.
LYMPHOMA & MYELOMA
Keith Stewart, MBChB, MBA
Mayo Clinic, Arizona
Elevate CLL R/R: Study of Acalabrutinib
(ACP-196) Versus Ibrutinib in Previously
Treated Subjects With High-Risk Chronic
Lymphocytic Leukemia (NCT02477696)
ACP-196 (Acalabrutinib) in Combination
With Pembrolizumab, for Treatment of
Hematologic Malignancies (KEYNOTE145)
(NCT02362035)
Study Design: Non-randomized, single-group
assignment, open-label safety/efficacy study
Study Start Date: February 2015
Estimated Study Completion Date: April 2021
Study Status: Currently recruiting participants
Estimated Enrollment: 187
Sponsor: Acerta Pharma BV
Acalabrutinib, also known as ACP-196, is a secondgeneration Bruton tyrosine kinase (BTK) inhibitor
designed to be a more potent and selective inhibitor of
BTK to avoid off-target side effects experienced with
other BTK inhibitors. When profiled against 395 human
kinases, ACP-196 was more selective than ibrutinib.
Two trials are determining the safety and efficacy of
ACP-196 in treating hematologic malignancies: The
first is examining whether acalabrutinib is as effective,
but less toxic, than Ibrutinib in treating previously
treated, high-risk chronic lymphocytic leukemia; the
second is evaluating the combination of acalabrutinib
plus the checkpoint inhibitor pembrolizumab in B-cell
malignancies.
A Phase 3, Randomized, Multicenter,
Double-Blind, Placebo-Controlled, 2-Arm,
Efficacy and Safety Study of NEOD001
Plus Standard of Care Versus Placebo Plus
Standard of Care in Subjects With Light
Chain (AL) Amyloidosis (NCT02312206)
Study Design: Randomized, parallel assignment,
double-blind safety/efficacy study
Study Start Date: February 2015
Estimated Study Completion Date: August 2018
Study Status: Currently recruiting participants
Estimated Enrollment: 236
Sponsor: Prothena Therapeutics Ltd.
NEOD001 is a humanized immunoglobulin G1 antibody,
directed against a cryptic epitope on amyloid fibrils.
NEOD001 specifically targets misfolded, light-chain
aggregates and amyloid deposits in patients with lightchain amyloidosis. This trial is evaluating the efficacy
of intravenous NEOD001 plus standard of care versus
placebo plus standard of care by assessing time to allcause mortality or cardiac hospitalization.
Study Design: Randomized, single-blind, parallelassignment study
Study Start Date: June 2015
Estimated Study Completion Date: June 2019
Study Status: Currently recruiting participants
Estimated Enrollment: 500
Sponsor: Acerta Pharma BV
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ASH Clinical News
December 2016