CLINICAL NEWS
Trial Roundup
ASH Clinical News’ Associate Editors select
clinical trials to keep an eye on.
LEUKEMIA
estimated study completion date:
BLEEDING DISORDERS
David Steensma, MD
Dana-Farber Cancer Institute
study status:
Alice Ma, MD
University of North Carolina School of Medicine
A Two-Arm Phase II Clinical Study of the
Clinical Efficacy and Safety of Tosedostat
in Patients With Myelodysplastic
Syndromes (MDS) After Failure of
Hypomethylating Agent-Based Therapy
This trial is investigating the combination of two
checkpoint inhibitors together with azacitidine (which
changes cell surface antigen expression and may augment immunogenicity of clonal cells) in MDS.
September 2021
Currently recruiting participants
estimated enrollment: 120
sponsor: MD Anderson Cancer Center
(NCT02452346)
LYMPHOMA & MYELOMA
study design:
Keith Stewart, MBChB, MBA
Mayo Clinic, Arizona
Non-randomized, open-label, singlegroup assignment safety/efficacy study
study start date: February 2015
estimated study completion date: September 2018
study status: Currently recruiting participants
estimated enrollment: 80
sponsor: Weill Medical College of Cornell University
Tosedostat, an oral inhibitor of aminopeptidase that
influences cellular protein clearance, has demonstrated activity in relapsed/refractory acute myeloid
leukemia (AML). This investigator-sponsored study
will examine its efficacy in myelodysplastic syndromes (MDS).
Dose Escalation of OXi4503 as Single
Agent and Combination With Cytarabine
w/ Subsequent Ph 2 Cohorts for AML and
MDS (NCT02576301)
study design:
Randomized, open-label, single-group
assignment safety/efficacy study
study start date: October 2015
estimated study completion date: October 2020
study status: Currently recruiting participants
estimated enrollment: 105
sponsor: Oxigene
The first phase of this study will investigate the
maximum tolerated dose of OXi4503 (combretastatin
A1 di-phosphate/CA1P), a dual-mechanism vascular disrupting agent (VDA) as a single agent and in
combination with intermediate-dose cytarabine in
subjects with relapsed/refractory AML or MDS. The
second phase will investigate the overall response
rate of OXi4503 in combination with intermediatedose cytarabine in two groups: patients with MDS
after failure of one prior hypomethylating agent (Arm
A) and patients with relapsed and refractory AML after treatment failure of up to one prior chemotherapy
regimen (Arm B).
Nivolumab and Ipilimumab With
5-Azacitidine in Patients With
Myelodysplastic Syndromes (NCT02530463)
study design:
Non-randomized, open-label, parallel assignment safety/efficacy study
study start date: September 2015
16
ASH Clinical News
Study Comparing Daratumumab,
Lenalidomide, and Dexamethasone With
Lenalidomide and Dexamethasone in
Participants With Previously Untreated
Multiple Myeloma (NCT02252172)
These two clinical trials look at two novel agents for
treating patients with hemophilia who have developed inhibitors. The first, called ACE910, is a bispecific
antibody against factor IX and factor X that binds and
holds them in the proper conformation to make the
tenase complex, in place of factor VIII. It is given as a
once-weekly subcutaneous injection. The second investigational agent is a small interfering RNA, or siRNA,
against antithrombin, and is designed to boost the
thrombogenic potential in patients with hemophilia A
and B with inhibitors.
A Phase III Study to Evaluate the
Efficacy, Safety, and Pharmacokinetics
of Prophylactic Emicizumab Versus No
Prophylaxis in Hemophilia A Patients
With Inhibitors (NCT02622321)
study design:
Randomized, open-label, parallel assignment efficacy study
study start date: February 2015
estimated study completion date: November 2022
study status: Currently recruiting participants
estimated enrollment: 730
sponsor: Janssen Research & Development, LLC
This study will compare the efficacy of daratumumab in
combination with lenalidomide and dexamethasone to
the combination of lenalidomide and dexamethasone,
measuring progression-free survival in participants with
newly diagnosed multiple myeloma who are not eligible
to receive high-dose chemotherapy and autologous
hematopoietic transplantation (autoHCT). The results of
this study will eventually be used to support the regulatory approval of front-line daratumumab.
Study of T Cells Targeting B-Cell
Maturation Antigen for Previously
Treated Multiple Myeloma (NCT02215967)
study design:
Open-label, single-group assignment
safety study
study start date: August 2014
estimated study completion date: October 2018
study status: Currently recruiting participants
estimated enrollment: 38
sponsor: National Cancer Institute
Immunotherapy with chimeric antigen receptor (CAR)
T-cell therapy has shown impressive early results in
certain hematologic malignancies. This study will
determine the safety of administering T cells expressing a B-cell maturation antigen CAR in patients with
previously treated myeloma.
study design:
Randomized, open-label, parallel assignment safety/efficacy study
study start date: November 2015
estimated study completion date: January 2018
study status: Currently recruiting participants
estimated enrollment: 70
sponsor: Hoffmann-La Roche
This multicenter study will evaluate the safety, efficacy, and pharmacokinetics of prophylactic ACE910
treatment in patients previously treated with episodic
or prophylactic bypassing agents. Patients will be
randomized in a 2:1 fashion to r eceive episodic treatment with prophylactic ACE910 (Arm A) or without
prophylactic ACE910 (Arm B). In Arm C, patients will
receive prophylactic ACE910 plus episodic treatment.
All patients will continue to receive standard-ofcare/background treatment with their usual episodic
bypassing agent therapy to treat breakthrough bleeds
as needed.
A Phase I Single-Ascending and MultipleAscending Dose, Safety, Tolerability,
and Pharmacokinetics Study of
Subcutaneously Administered ALNAT3SC in Healthy Adult Volunteers and
Hemophilia A or B Patients (Moderate or
Severe Hemophilia) (NCT02035605)
study design:
Randomized, single-blind, parallel
assignment safety study
study start date: January 2014
estimated study completion date: April 2017
study status: Currently recruiting participants
estimated enrollment: 72
sponsor: Alnylam Pharmaceuticals
This study will determine the safety, tolerability, and
pharmacokinetics of the siRNA ALN-AT3SC in healthy
volunteers and patients with hemophilia A or B.
June 2016