CURE's Clinical Trial Corner: December 2019

As the cancer treatment landscape continues to grow, patients and their caregivers should be aware of the various clinical trials currently being conducted — and ones they can possibly join.

Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

The first patient has been enrolled in the pivotal phase 2/3 clinical study for JZP-458, which is being conducted in collaboration with the Children's Oncology Group (COG). The single-arm, open-label, multicenter, dose confirmation and confirmatory trial is designed to evaluate the safety, tolerability and efficacy of JZP-458 as a potential treatment for pediatric and adult patients with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) who are hypersensitive to E. coli-derived asparaginases.

This study is expected to enroll patients in approximately 60 COG institutions in the U.S. and Canada.

RET-Mutant Medullary Thyroid Cancer

Eli Lilly and Company have launched the randomized phase 3 LIBRETTO-531 clinical trial — designed to evaluate selpercatinib (LOXO-292) compared with physician’s choice of Cabometyx (cabozantinib) or Caprelsa (vandetanib) for the initial treatment of treatment-naïve patients with RET-mutant medullary thyroid cancer. For patients randomized to the control arm (physician’s choice therapy), crossover is allowed at progression.

The primary endpoints of the study will be progression-free survival (the time from treatment to disease progression or worsening), treatment failure-free survival (the absence of relapse or non-relapse mortality or the addition of another systemic therapy), overall survival, overall response rate and duration of response. The trial will enroll 400 patients with advanced or metastatic RET-mutant medullary thyroid cancer who have received no prior systemic therapy for metastatic disease.

Waldenström’s Macroglobulinemia

X4 Pharmaceuticals, Inc. initiated a phase 1b trial of mavorixafor (X4P-001) in combination with Imbruvica (ibrutinib) for the treatment of Waldenström’s macroglobulinemia, a rare form of non-Hodgkin’s lymphoma. The multicenter, open-label, dose-escalation, clinical trial is designed to assess the safety and tolerability of mavorixafor in combination with Imbruvica in patients with Waldenström’s macroglobulinemia who have acquired a “gain of function” mutation in CXCR4 in addition to the MYD88 mutation.

The clinical trial, which is being conducted as part of a collaboration with The Leukemia & Lymphoma Society, is expected to enroll approximately 12-18 patients.