PEPN2111: Study of CBL0137 in Patients with Relapsed or Refractory Solid Tumors and Lymphoma

Brief Summary

This phase 1/2 trial evaluates the novel small-molecule inhibitor CBL0137 in treating patients with solid tumors or relapsed/refractory lymphoma. CBL0137 is in a class of anti-cancer agents called curaxins. CBL0137 targets the FACT complex to inhibit over-expression of proteins related to aggressive cancers.  Preclinical studies suggest CBL0137 has anti-tumor activity against certain pediatric cancer cell lines and is active in solid tumor models.

Patients will receive up to 17 cycles of CBL0137 intravenously, with follow-up occurring regularly until 60 months after completion of study treatment.

Primary Objectives

• To estimate the maximum tolerated dose and/or recommended Phase 2 dose of CBL0137 in children with recurrent or refractory solid tumors and lymphoma
• To preliminarily determine antitumor effects of CBL0137 in children with progressive/recurrent DIPG and other H3 K27M-mutant diffuse midline gliomas
• To preliminarily determine antitumor effects of CBL0137 in children, adolescents and young adults with osteosarcoma

Eligibility

Inclusion criteria include:

• 12 months to 21 years old with diagnosis of:
  • Relapsed or refractory solid tumor or lymphoma (including CNS tumors) OR
  • Progressive or recurrent DIPG or other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy
• 12 months to 30 years old with diagnosis of relapsed or refractory osteosarcoma
• Adequate bone marrow and organ function
• Recovered fully from prior anti-cancer therapy
• Karnofsky or Lansky score of at least 50%
• No prior exposure to CBL0137

Exclusion Criteria include:

• Pregnant or breastfeeding
• Currently receiving other anti-cancer agents (except leukemia patients receiving hydroxyurea)
• Currently receiving another investigational drug
• Known peripheral vascular disease
• Uncontrolled infection
• Prior solid organ transplant

Study Sites

St. Jude Children’s Research Hospital
Memphis, Tennessee

Collaborating sites in the U.S.

About this study

This is a clinical trial of a cancer-fighting medicine called CBL0137. Doctors want to learn how this medicine works in young patients with cancer that has come back after treatment (relapsed) or did not get better after treatment (refractory).

CBL0137 is experimental. This means it has not been proven to work in children with these types of cancer. Researchers want to learn more about CBL0137 because it seems to work against cancer in test tubes and animals. CBL1037 has been used in adults with cancer, but there is a lot we do not know about it yet.

In this study, you will be given CBL0137 through a vein. There are 17 cycles of therapy. Each cycle lasts 21 days. The entire study treatment will last about 12 months.

Purpose of this clinical trial

The main purpose of this study is to find out the highest, safest dose of CBL0137 that can be given to children, adolescents and young adults with solid tumors or lymphoma.

Eligibility overview

• 12 months to 21 years old with diagnosis of:
  • Relapsed or refractory solid tumor or lymphoma (including CNS tumors) OR
  • Progressive or recurrent DIPG or other H3 K27M-mutant diffuse midline gliomas previously treated with radiation therapy
• 12 months to 30 years old with diagnosis of relapsed or refractory osteosarcoma