New drug cocktail aims to fight tough childhood cancer

NCT ID NCT02298348

Ongoing Disease control Sponsor: New Approaches to Neuroblastoma Therapy Consortium Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests a combination of three drugs—sorafenib, cyclophosphamide, and topotecan—in children and young adults with neuroblastoma that has relapsed or not responded to treatment. The main goal is to find the highest safe dose of sorafenib when given with the two chemotherapy drugs. The study involves 18 participants and focuses on safety and side effects, not yet on curing the disease.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Sorafenib (a targeted cancer drug) combined with cyclophosphamide and topotecan (chemotherapy drugs)
What this could lead to: If successful, this could point toward a new treatment option for children with neuroblastoma that has come back or not responded to standard therapy.
What could go wrong: This is a very early (Phase 1) and small trial (18 patients) focused on finding a safe dose, not on proving effectiveness. The combination may cause significant side effects and may not work for everyone.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

neuroblastoma Recurrence

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

NEUROBLASTOMA

Contacts and locations

Locations

C.S Mott Children's Hospital

Ann Arbor, Michigan, 48109, United States
Children Hospital of Colorado

Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States
Children's Hospital Los Angeles

Los Angeles, California, 90027-0700, United States
Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, 98105, United States
Childrens Hospital Boston, Dana-Farber Cancer Institute.

Boston, Massachusetts, 02115, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States
Cook Children's Healthcare System

Fort Worth, Texas, 76104, United States
Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
UCSF Comprehensive Cancer Center

San Francisco, California, 94143, United States
University of Chicago, Comer Children's Hospital

Chicago, Illinois, 60637, United States
University of North Carolina

Chapel Hill, North Carolina, 27599, United States

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