Trial Roundup: PRTX-100 for Immune Thrombocytopenia, APR-246 for Myeloid Neoplasms, and more

ASH Clinical News’ Associate Editors select clinical trials to keep an eye on in leukemia, bleeding disorders, and lymphoma & myeloma.

LEUKEMIA

David Steensma, MD
Dana-Farber Cancer Institute

A Phase Ib/II Study to Evaluate the Safety and Efficacy of APR-246 in Combination With Azacitidine for the Treatment of TP53 Mutant Myeloid Neoplasms (NCT03072043)

  • Study Design: Non-randomized, crossover-assignment, open-label study
  • Study Start Data: May 2017
  • Estimated Study Completion Date: May 2020
  • Study Status: Currently recruiting participants
  • Estimated Enrollment: 60
  • Sponsor: H. Lee Moffitt Cancer Center and Research Institute

APR-246 is a methylated derivative and structural analog of PRIMA-1 that may reactivate mutant p53 protein to induce tumor cell death. This study will determine the maximum tolerated dose of APR-246 (administered with azacitidine) in patients with TP53-mutant myelodysplastic syndromes, acute myeloid leukemia (AML), myeloproliferative neoplasms, or chronic myelomonocytic leukemia. In the second phase of the study, investigators will determine the eight-month overall survival (OS) for patients treated with the recommended phase II dose of APR-246 and azacitidine.

A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment-Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy (NCT02993523)

  • Study Design: Randomized, parallel assignment,
    double-blind study
  • Study Start Date: February 2017
  • Estimated Study Completion Date: January 2021
  • Study Status: Currently recruiting participants
  • Estimated Enrollment: 400
  • Sponsor: AbbVie

This phase III, double-blind, randomized, placebo-controlled trial will evaluate whether adding venetoclax to azacitidine improves response, compared with azacitidine alone, in adult patients with treatment-naïve AML. Primary endpoints include rates of complete remission (CR) and OS at six months post-randomization; secondary endpoints include time to CR, event-free survival, and patient-reported fatigue.

BLEEDING DISORDERS

Alice Ma, MD
University of North Carolina School of Medicine

A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment-Naïve Subjects With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy (NCT02993523)

  • Study Design: Single-group assignment, open-label study
  • Study Start Date: November 2015
  • Estimated Study Completion Date: May 2018
  • Study Status: Currently recruiting participants
  • Estimated Enrollment: 30
  • Sponsor: Protalex, Inc.

In mouse models of ITP, the investigational agent PRTX-100 (a highly purified form of staphylococcal protein A) was shown to improve platelet levels, leading the U.S. Food and Drug Administration to grant the agent orphan drug designation. In this dose-escalation phase of the study, patients with chronic/persistent ITP will be assigned consecutively to receive four weekly infusions of either 3, 6, 12, 18, or 24 μg/kg to determine the safety profile of PRTX-100. Secondary endpoints include platelet response, time until response, and change in platelet counts from baseline to 11 months of treatment.

End of EXTEND: Discontinuation of Medication for Patients With Immune Thrombocytopenia (NCT01386723)

  • Study Design: Prospective, observational study
  • Study Start Date: June 2011
  • Estimated Study Completion Date: June 2019
  • Study Status: Currently recruiting participants
  • Estimated Enrollment: 20
  • Sponsor: Weill Cornell Medicine

The objective of this study is to determine whether patients with immune thrombocytopenia (ITP) who have discontinued eltrombopag and have not been immediately transitioned to additional treatment can attain a stable platelet count for four to eight weeks after treatment discontinuation. Researchers will also measure how long a potential response lasts.

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