A phase 1 trial of venetoclax in children and young adults with relapsed or refractory cancers, including non-Hodgkin lymphomas

This trial is testing a treatment called venetoclax in children and young people with cancer, including non-Hodgkin lymphoma, that has come back or not responded to previous treatment. In this summary, we focus on information that is relevant to people who have lymphoma.


Purpose of trial

The main aim of this trial is to find out if venetoclax is safe to use in children and young adults and, if so, the best dose to use.


Treatments

Everybody who takes part in this trial is treated with venetoclax. You have it as a tablet or liquid medicine that you take by mouth once a day. If your consultant thinks you need it, you will also have chemotherapy.

Venetoclax is a type of targeted drug called a BCL-2 inhibitor.

  • Cells have a natural lifespan. When they get old, they die and are replaced by new cells. This process is usually kept carefully in balance.
  • B-cell lymphoma-2 (BCL-2) is a protein that can block the natural process that makes cells die when they should. Some cancer cells make a lot more BCL-2 than healthy cells, which helps them stay alive longer than they should.
  • BCL-2 inhibitors block the BCL-2 protein. This activates the process of natural cell death.

Who can enter

Your consultant can give you advice on whether you might be suitable for this trial.

Around 165 people are needed for this trial, including 25 with non-Hodgkin lymphoma.

You may be able to enter if:

  • You have cancer, including non-Hodgkin lymphoma.
  • You have previously had at least one course of treatment for your lymphoma.
  • You are well enough to take part in the trial.
  • Your blood and other health test results are satisfactory.
  • You and your partner are willing to use reliable contraception if there is any chance of you or your partner becoming pregnant.
  • You are under 25.

You won’t be able to enter if:

  • You have any health problems that your doctor thinks might make it unsafe for you to have the trial treatment.
  • You still have significant side effects from your previous treatment.
  • You have lymphoma in your central nervous system (CNS; brain and spinal cord) that needs treatment with radiotherapy.
  • You’ve had chemotherapy, radiotherapy, or any targeted treatment for your cancer in the last 2 weeks.
  • You’ve had antibody therapy in the last 30 days.
  • You’ve had CAR T-cell therapy in the last 30 days.
  • You’ve had steroid treatment for your lymphoma in the last 5 days.
  • You’ve had a stem cell transplant in the last 100 days (or more than 100 days ago if you have active graft-versus-host disease or are still taking drugs to dampen your immune system).
  • You’ve had drugs that might interfere with the study treatment in the last week.
  • You have an uncontrolled infection.
  • You have a condition that means you can’t absorb food properly.
  • You have COVID-19.
  • You are pregnant or breastfeeding.

Further information

More information about this trial is available at https://clinicaltrials.gov/ct2/show/NCT03236857