New drug cocktail shows promise for kids with resistant cancers
NCT ID NCT01282697
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tested a combination of two drugs—rapamycin and irinotecan—in 42 children and young adults (ages 1–21) with solid tumors that did not respond to standard treatments. The main goal was to find the safest dose and understand side effects. The approach aims to block blood vessel growth in tumors and stop their progression.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- rapamycin and irinotecan
- What this could lead to
- If this works, it could point toward a new treatment option for children with solid tumors that haven't responded to standard therapies.
- What could go wrong
- This is a very early phase I trial with only 42 children, focused on safety and dosing—not yet on effectiveness. The combination may cause significant side effects or fail to shrink tumors.
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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42 people
The number who actually took part.
- Started
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Apr 2011
- Finished
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Feb 2013
- Lead sponsor
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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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1 year to 21 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: * Age \>= 1 year old and =\< 21 years old; * Refractory solid tumors, histologically proven at diagnosis (no additional biopsy needs to be performed for the purpose of the study); * Relapsed or refractory solid tumors after standard treatment or phase II, III-IV clinical trials treatment have failed; * Karnofsky or Lansky status \>= 70%; * Life expectancy \>= 8 weeks; * No chemotherapy / radiotherapy within 4 weeks before entry into the study; * Adequate biological parameters : * Absolute neutrophil count \>= 1.0 x 109/L; * Platelet count \>= 100 x 109/L; * Hemoglobin \>= 8 mg/dL; * Total bilirubine =\< 1.5 ULN; * Transaminases =\< 2.5 ULN (=\< 5 ULN in case of liver metastases); * Creatinine clearance (Cockroft) \>= 70 mL/min/1.73 m2; * Normal coagulation profile with prothrombin \>= 70%, TCA =\< 35 and fibrinogen \>= 2 g/L; * Patients with 1 to 3 previous therapeutic lines are eligible; * No current grade \>= 2 organ toxicity based on NCI-CTCAE version 3.0; * All patients with reproductive potential must have an effective method of birth control while on study; * Negative pregnancy test in females when indicated; * Informed written consent signed by patients or their parents or legal guardians; * Patient who was informed of the results of prior medical consultation; * Patient having a social insurance. Exclusion Criteria: * Patient with a constitutional anomaly of coagulation and/or of hemostasis (type hemophilia, von Willebrand disease, congenital clotting factor deficit, platelet disorder), exposing them to increased risk of bleeding; * Pre-treatment with a mTOR inhibitor; * Other simultaneous malignancy; * Concurrent administration of any other anti-tumour therapy; * Known hypersensitivity or contraindication to study drugs or ingredients; * Severe concomitant disease (e.g. infection disease); * Patient unable for medical follow-up; * Pregnancy and/or lactation; * Patient included in another clinical drug trial; * Patient taking drugs interfering with pharmacology of rapamycin and/or irinotecan (e.g. drugs interfering with CYP3A4); * Patient under judicial protection.
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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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CHU La Timone
Marseille, 13005, France
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CHU Mère-Enfants
Nantes, 44093, France
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Centre Oscar Lambret
Lille, 59020, France
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Hôpital des Enfants
Toulouse, 31059, France
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Hôpital des Enfants - Groupe Hospitalier Pellegrin
Bordeaux, 33076, France
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Hôpitaux Universitaires de Strasbourg
Strasbourg, 67098, France
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Institut Curie
Paris, 75005, France
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Institut Gustave Roussy
Villejuif, 94805, France
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Institut Hémato-Oncologie Pédiatrique (IHOP)
Lyon, 67008, France
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