T Cell Therapy for Previously Treated Multiple Myeloma, Emicizumab in Hemophilia A Patients With Inhibitors, and more

LEUKEMIA

David Steensma, MD
Dana-Farber Cancer Institute

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 (NCT02452346)

  • study design: Non-randomized, open-label, single-group 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 intermediate-dose 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
  • estimated study completion date: September 2021
  • study status: Currently recruiting participants
  • estimated enrollment: 120
  • sponsor: MD Anderson Cancer Center

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.


LYMPHOMA & MYELOMA

Keith Stewart, MBChB, MBA
Mayo Clinic, Arizona

Study Comparing Daratumumab, Lenalidomide, and Dexamethasone With Lenalidomide and Dexamethasone in Participants With Previously Untreated Multiple Myeloma (NCT02252172)

  • 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.


BLEEDING DISORDERS

Alice Ma, MD
University of North Carolina School of Medicine

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 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 receive 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-of-care/background treatment with their usual episodic bypassing agent therapy to treat breakthrough bleeds as needed.

A Phase I Single-Ascending and Multiple-Ascending Dose, Safety, Tolerability, and Pharmacokinetics Study of Subcutaneously Administered ALN-AT3SC 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.

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