NCI, The Leukemia & Lymphoma Society and the Children’s Oncology Group plan to develop a global precision medicine clinical trial for children with acute leukemia.
The new study, called LLS PedAL (Pediatric Acute Leukemia), is a master clinical trial that will test simultaneously multiple targeted therapies for children who experience a relapse of their acute leukemia, experienced by approximately 40% of children with AML, and 20% of children with high-risk acute lymphoblastic leukemia.
The study will take place at more than 200 sites that are part of the NCI-supported COG network of children’s hospitals, including those in the U.S., Australia, New Zealand, and Canada. The LLS PedAL team will also collaborate with other overseas partners in the UK and EU to implement the trial in those regions. Through these established clinical trial infrastructures, nearly every child in these regions who experiences a relapse of acute leukemia will have access to the trial.
LLS PedAL is a key component of The LLS Children’s Initiative, the society’s $100 million multi-year endeavor to attack pediatric blood cancer from every direction: research grants to advance novel treatments for children, enhanced free education and support services for children and their families, and new policy and advocacy efforts.
LLS led the Beat AML Master Clinical Trial, a precision medicine trial for adults with acute myeloid leukemia. The trial tested novel targeted therapies and identified disease subtypes based on specific biomarkers. Then, researchers matched them with treatments best suited for their diagnosis. A similar model will be used in the LLS PedAL trial, which will match children whose acute leukemia has returned with targeted therapies.
Before the LLS PedAL trial can launch, LLS must secure approval from FDA to begin testing therapies in patients. LLS will be responsible for regulatory submissions to the FDA and will be the Investigational New Drug holder. LLS plans to submit its IND application to the FDA later this year.
The NCI Pediatric Central Institutional Review Board will review all protocols for all of the sites in the COG network before the trial begins. The COG Data Safety Monitoring Committee will provide oversight once the trial is underway.
“Many targeted therapies have been approved to treat adults with acute leukemia over the past decade, but few have been sufficiently studied in children to demonstrate their benefit. This study will give us the opportunity to learn what new targeted therapies are effective for children with acute leukemias,” E. Anders Kolb, Nemours Center for Cancer and Blood Disorders, chair of the COG Myeloid Disease Committee, and LLS Pedal co-chair, said in a statement.
The LLS PedAL trial will launch with three novel therapies to treat children with relapsed acute leukemia, with plans to add additional treatments as they become available. The study will ensure that every child with relapsed AML and many with ALL will be screened to identify their disease subtype and matched to the most appropriate treatment. Some of these patients will participate in LLS PedAL substudies, while others will be referred to other treatments, including other open clinical trials based on discussions with their physicians.
The LLS PedAL trial will include a technology platform to enable participating institutions to consolidate, share and analyze data about pediatric blood cancer patients using standardized terminology. The data will help doctors better understand how children will respond to novel therapies, and the underlying causes of resistance or relapse.
