Contract research organization (CRO) PRA Health Sciences is partnering with the Leukemia and Lymphoma Society (LLS) to launch a first-of-its-kind global master clinical trial, designed to develop novel treatments for pediatric patients with relapsed acute leukemia.
The Pediatric Acute Leukemia (PedAL) clinical trial will simultaneously test multiple targeted therapies for the children whose acute leukemia relapses; approximately 40% of children with acute myeloid leukemia (AML) and 20% of children with high-risk acute lymphoblastic leukemia (ALL) experience relapses.
Brandon Early, vice president of project management at PRA, told Outsourcing-Pharma that the purpose of the LLS PedAL master clinical trial is to “fundamentally change” treatment options and methods for pediatric acute leukemia patients.
“The way cancers behave in children and how a child responds to therapy are profoundly different from that of adults,” Early said. “Thanks to the power of precision medicine, which focuses on giving the right treatment to the right patient at the right time, we are seeing a much-needed revolution in cancer treatment.”
Unfortunately, Early pointed out, progress in treatment development for pediatric cancers is not keeping pace with therapies for adult oncological patients.
“New and better treatments are reaching adults at a dizzying pace, while only four cancer treatments have been approved for first use in children over the past four decades," he explained. "For too long, treatment for pediatric acute leukemia has followed a one-size-fits-all approach.; this is where LLS PedAL comes in – this first-of-its kind global precision medicine master clinical trial will test multiple targeted therapies simultaneously at up to 200 clinical sites worldwide.”
The study will occur at the worldwide sites that are part of the NCI-supported Children's Oncology Group (COG) network of children's hospitals, including hospitals in the US, Australia, New Zealand and Canada; the trial team also will work with partners in the UK and EU to implement the trial in those regions. According to PRA, nearly every child in the regions whose acute leukemia relapses will have access to the trial.
The PedAL trial will commence with a number of novel therapies to treat relapsed acute leukemia, with plans to add additional treatments as they become available. The goal is to make it possible for every child with relapsed AML and many with ALL to be screened, in order to identify disease subtype and match them to the best targeted treatment.
PRA will contribute to the trial by managing various aspect of the trial, including coordinating the sites, ensuring protocols are executed properly, providing supporting technology, and monitoring data quality and safety reporting. The CRO also will offer guidance for submissions to the US Food and Drug Administration (FDA) and other regulatory agencies.
“Trials of this complexity and scope require great collaboration and are what we’re going to need in order to see improvement in survival for these highest risk patients,” Early explained. “This trial is unique because a CRO has never partnered with a nonprofit for a pediatric leukemia trial of this magnitude.”
In 2017, the US Congress passed the Research to Accelerate Cures and Equity (RACE) for Children Act into law, which went into effect on August 18. The law requires pharma companies testing new molecularly targeted compounds for adult cancers to also evaluate them for children's cancers.