NCI Announces a Pediatric MATCH Trial for Childhood Cancer

Sep 19, 2014

NCI Molecular Analysis for Therapy Choice Program (MATCH) & Pediatric MATCH

NCI will be launching two new precision medicine trials – Molecular Analysis for Therapy Choice Program (MATCH) and Pediatric MATCH.


The NCI MATCH Trial will include small, phase II trials that will enroll adults with advanced solid tumors and lymphomas—including up to 25% with rare cancers—whose tumors are no longer responding to standard therapy and have begun to grow.

Biopsies from tumors from as many as 3,000 patients will undergo next-generation DNA sequencing to identify individuals whose tumors have genetic abnormalities that may respond to selected targeted drugs. These genetic mutations have been chosen based on the availability of targeted drugs, including those that have been approved by the Food and Drug Administration (FDA) for another indication or that are still investigational, but have shown some efficacy against tumors with one of the genetic alterations.

As many as 1,000 patients will then be assigned to one of the phase II trials, each involving approximately 30 patients, based not on their type of cancer but on the genetic abnormality that is thought to be driving their cancer. In each phase II trial, patients will be treated with one of approximately 20 to 25 drugs initially available for the trial that target the genetic abnormalities that are being tested for in this study. The NCI MATCH Trial is a master protocol, meaning that new treatments can be brought in over time.

Patients’ tumors often have different genetic abnormalities that could be causing their disease to progress. Decisions about which trial/agent patients will be assigned to will be based on predetermined decision rules that dictate treatment according to the genetic abnormality present in the tumor.

The primary endpoint for the MATCH trials will be tumor response, but progression-free survival will also be of interest. Patients whose cancers progress during the first assigned treatment may be able to go on another MATCH trial arm if they have a second actionable molecular target in their tumors. In addition, any patient whose cancer initially shrinks and then progresses during the trial will be eligible to have their tumors re-biopsied and, if they have a genetic change that is targeted by another drug being tested in MATCH, they may be eligible to enroll in one of the other phase II MATCH trials.

Once patients come off the study, their treating physician will receive a report on the results of the tumor DNA analysis, in the hope that the information may be useful for future treatment choices.

The NCI MATCH Trial will be open to all members of the four NCTN Adult Groups. The ECOG-ACRIN Cancer Research Group will lead the study, along with NCI.

Pediatric MATCH

The NCI MATCH Trial will also have a pediatric counterpart that will enroll children with advanced cancers that have progressed on standard therapy.

As in the adult MATCH Trial, DNA sequencing will be used to identify children whose tumors have a genetic abnormality for which either an approved or investigational targeted therapy exists. NCI is working with numerous pharmaceutical companies to make the same drugs available for Pediatric MATCH that will be offered in the adult MATCH trials.

Pediatric MATCH provides a tremendous opportunity to test molecularly targeted therapies in children with advanced cancers who have few other treatment options. With the genomic data captured in the trials, it will also produce an invaluable resource for studying the genetic basis of pediatric cancers.

Pediatric MATCH, which will be led by the NCI-funded Children’s Oncology Group, is still under development and details on a timetable for launch or patient enrollment are not yet available.