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Name of the Trial

Phase I Study of Trabectedin in Pediatric Patients With Relapsed or Refractory Solid Tumors (NCI-07-C-0054). See the protocol summary.

Principal Investigators

Dr. Elizabeth Fox and Dr. Meredith Chuk (Lead Associate Investigator), NCI Center for Cancer Research

Why This Trial Is Important

New chemotherapy treatments for childhood cancers are often based on drugs developed to treat adult cancers. However, adults and children may not always react the same way to the same drugs. Because children's bodies are undergoing rapid growth and development, pediatric patients may be adversely affected in ways not seen in adult patients. It is critically important, therefore, to conduct early-phase clinical trials to find the best dose of a new drug for children with cancer and to determine any side effects in these young patients.

Trabectedin was originally derived from the Mangrove tunicate, a species of sea squirt. (Photo courtesy of Kathy Hill, Smithsonian Marine Station at Fort Pierce)

A new drug called trabectedin (Yondelis, ET-743), which was originally obtained from marine creatures known as sea squirts, is being developed for the treatment of adult patients with soft tissue sarcoma, ovarian cancer, and other solid tumors. This drug is now made in the laboratory and appears to kill cells by damaging their DNA and by interfering with DNA repair. In laboratory and animal testing, trabectedin was shown to be effective in killing cancer cells from several different types of solid tumors that occur in children, leading researchers to pursue its development for use against these pediatric cancers.

In this trial, children with solid tumors that have either recurred or have not responded to previous treatment will be treated with trabectedin. The patients will receive trabectedin by intravenous infusion over a 24-hour period once every 21 days. The doses will be increased in consecutive groups of patients until the maximum tolerated dose is reached. The side effects of trabectedin will also be determined.

"Although this drug has undergone limited testing in pediatric patients previously, using a shorter infusion schedule, we need to establish the maximum tolerated dose with this new 24-hour infusion schedule, which is the recommended schedule for adults," said Dr. Fox. "In addition, most research on trabectedin use in children focuses primarily on patients with sarcoma. We hope to also recruit patients with non-sarcoma cancers, so we can explore the drug's potential in a broader range of childhood cancers."

For More Information

See the lists of entry criteria and trial contact information or call the NCI Clinical Trials Referral Office at 1-888-NCI-1937. The call is toll free and confidential.

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