PEPN1812 : Study of Flotetuzumab in Children with Relapsed or Refractory Acute Myeloid Leukemia

Brief Summary

This Phase I study will evaluate flotetuzumab in pediatric patients with relapsed/refractory AML. Flotetuzumab is a bispecific antibody designed to target CD3 and CD123. 

Patients will receive a continuous infusion of flotetuzumab due to the favorable toxicity and efficacy profile of this administration schedule in adults with AML. Patients may receive up to a total of 6 cycles of flotetuzumab unless they are unable to achieve a partial response (PR) after Cycle 2 or are found to have progressive disease at any time point. 

Primary Objectives

• To assess the safety and tolerability of flotetuzumab administered by continuous IV infusion to pediatric patients younger than 21 years of age with relapsed or refractory AML
• To estimate the maximum tolerated dose (MTD) and/or recommended Phase II dose of flotetuzumab administered by continuous IV infusion to pediatric patients younger than 21 years of age with relapsed or refractory AML

Eligibility Criteria

Inclusion criteria include:

• Younger than 21 years of age
• Diagnosed with recurrent or refractory acute myeloid leukemia (AML)
• Weigh more than 17kg 
• Fully recovered from the acute toxic effects of all prior anti-cancer therapy
• Adequate bone and marrow function

Exclusion Criteria include:

• No prior therapy with a CD123 directed antibody or CD123 directed CAR T cells
• Pregnant or breastfeeding
• Currently receiving another investigational drug
• Currently receiving other anti-cancer agents are not eligible [except hydroxyurea, which may be continued until 24 hours prior to start of protocol therapy].
• Receiving cyclosporine, tacrolimus or other agents to treat graft-versus-host disease post bone marrow transplant are not eligible for this trial.

Study Sites

St. Jude Children’s Research Hospital 
Memphis, Tennessee

Collaborating sites in the U.S.

About this study

This study looks at how well the experimental drug flotetuzumab works when given to children and young adults with relapsed or refractory acute myeloid leukemia. Flotetuzumab is experimental because it has not been proven to work in young patients with this type of AML. Although flotetuzumab has been tested in adults, it has not been approved for use by the FDA for adults or for children.

We are using flotetuzumab because it seems to work against cancer in test tubes and animals. This is called a Phase I study because the goal is to find the highest dose of flotetuzumab that we can give safely. If there is no improvement in your leukemia after two cycles of treatment, your treatment with flotetuzumab will be stopped.

Purpose of this clinical trial

The main goal of this study is to find the highest dose of flotetuzumab that we can give safely.

Eligibility overview

• Younger than 21 years of age
• Diagnosed with recurrent or refractory acute myeloid leukemia (AML)
• Weigh more than 17kg
• Fully recovered from the acute toxic effects of all prior anti-cancer therapy