New drug cocktail aims to tackle childhood cancers that Won't quit
NCT ID NCT03611595
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested a combination of two drugs, cabozantinib and 13-cis-retinoic acid, in 15 children whose solid tumors had come back or were not responding to standard treatments. The main goal was to find the safest dose and understand side effects. The study did not aim to cure the cancer, but to see if this drug pair could be a future option for controlling 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
- Cabozantinib and 13-cis-retinoic acid (isotretinoin)
- What this could lead to
- If successful, this could point toward a new treatment option for children with hard-to-treat solid tumors that have come back or not responded to standard therapy.
- What could go wrong
- This is a very early phase I trial with only 15 children, focused on safety and dosing, not on effectiveness. The drug combination may not shrink tumors or may cause significant side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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15 people
The number who actually took part.
- Started
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Aug 2018
- Finished
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Mar 2026
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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2 to 26 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. Patients must have had histologic verification of a solid tumor, including tumors of the CNS, at the time of initial diagnosis or relapse, with disease that has progressed on standard therapy, relapsed after standard therapy, or for which no standard curative therapy is known 2. Patients must have documentation of either measurable or evaluable disease within 4 weeks of onset of study therapy 3. Performance Status - Lansky play or Karnofsky score of ≥40 4. Prior Therapy: Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to study enrolment with the exception of hematologic parameters (absolute neutrophil count, hemoglobin, platelet count), which need to have recovered to meet eligibility criteria 5 Steroids are permitted for control of emesis and for symptom management in patients with intracranial metastases. However, patients with known CNS disease or CNS metastases who require increasing doses of steroids are not allowed in the study. Patients MUST be on a stable or decreasing steroid dose for greater than or equal to 1 week prior to start of study therapy. 6\. Female subjects of childbearing potential must agree to use an adequate method of contraception for the course of the study. Male subjects must agree to use an adequate method of contraception for the course of the study Exclusion Criteria: 1. Evidence of severe or uncontrolled systemic disease 2. Cardiac Disease 3. Blood pressure \>95th percentile for age (either systolic or diastolic) or \>140/90 for patients \>18 years of age and uncontrolled by oral medication at onset of study therapy 4. Women who are currently pregnant or breastfeeding. 5. Prior therapy with cabozantinib at any time. 6. Major surgery within 8 weeks before starting study therapy. 7. Prior treatment with allogeneic stem cell transplantation or total body irradiation (TBI) 8. Therapeutic anticoagulation with heparin, LMWH , or any other agents are not allowed in subjects with intracranial tumors/metastatses. 9. Concomitant anticoagulation at therapeutic doses with oral anticoagulants (eg, warfarin, direct thrombin and Factor Xa inhibitors) or platelet inhibitors (eg, clopidogrel) in patients without primary or metastatic CNS tumors 10. The subject has experienced any of the following: 1. clinically-significant GI bleeding within 6 months before the first dose of study treatment; 2. any other signs indicative of pulmonary hemorrhage within 3 months before the first dose of study treatment. 11. The subject has radiographic evidence of cavitating pulmonary lesion(s) and/or the subject has tumor invading any major blood vessels; 12. The subject has evidence of tumor invading the GI tract (esophagus, stomach, small or large bowel, rectum or anus), or any evidence of endotracheal or endobronchial tumor within 28 days before the first dose of cabozantinib 13. thromboembolic event requiring therapeutic anticoagulation within 6 months of onset of study treatment 14. GI disorders particularly those associated with a high risk of perforation or fistula formation 15. Inability to swallow intact tablets
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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.
Contacts and locations
Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
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Rady Children's Hospital - San Diego
San Diego, California, 92123, United States
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