Evaluating Acalabrutinib for Hematologic Malignancies, Minimizing Menorrhagia in Women With Type 1 Von Willebrand Disease, and more

LEUKEMIA

David Steensma, MD
Dana-Farber Cancer Institute

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)

  • Study Design: Non-randomized, parallel-assignment, open-label safety/efficacy study
  • Study Start Date: August 2016
  • Estimated Study Completion Date: 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 GMI-1271, 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.


BLEEDING DISORDERS

Alice Ma, MD
University of North Carolina School of Medicine

Minimize Menorrhagia in Women With Type 1 Von Willebrand Disease (VWDMin) (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)

  • Study Design: Randomized, single-blind, parallel-assignment study
  • Study Start Date: June 2015
  • Estimated Study Completion Date: June 2019
  • Study Status: Currently recruiting participants
  • Estimated Enrollment: 500
  • Sponsor: Acerta Pharma BV

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 second-generation 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 light-chain amyloidosis. This trial is evaluating the efficacy of intravenous NEOD001 plus standard of care versus placebo plus standard of care by assessing time to all-cause mortality or cardiac hospitalization.

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