New drug cocktail aims to fight tough childhood cancer
NCT ID NCT02298348
First seen Mar 26, 2026 · Last updated Jun 22, 2026 · Updated 7 times
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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C.S Mott Children's Hospital
Ann Arbor, Michigan, 48109, United States
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Children Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Children's Hospital Los Angeles
Los Angeles, California, 90027-0700, United States
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Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, 98105, United States
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Childrens Hospital Boston, Dana-Farber Cancer Institute.
Boston, Massachusetts, 02115, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229-3039, United States
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Cook Children's Healthcare System
Fort Worth, Texas, 76104, United States
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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UCSF Comprehensive Cancer Center
San Francisco, California, 94143, United States
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University of Chicago, Comer Children's Hospital
Chicago, Illinois, 60637, United States
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University of North Carolina
Chapel Hill, North Carolina, 27599, United States
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.
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.