Can a Two-Drug combo outsmart returning childhood cancers?
NCT ID NCT00918320
First seen Aug 19, 2026 · Last updated Aug 20, 2026 · Updated 1 time
Summary
This phase 2 trial is testing whether combining two chemotherapy drugs—temozolomide and topotecan—can help children with neuroblastoma, brain tumors, or other solid tumors that have come back or not responded to standard treatment. Participants receive temozolomide by mouth and topotecan by IV once daily for five days per cycle. The main goal is to see how often tumors shrink or disappear, while also tracking side effects and how long any benefit lasts.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- A combination of two chemotherapy drugs: temozolomide (taken by mouth) and topotecan (given by IV infusion).
- What this could lead to
- If effective, this drug combination could offer a new treatment option for children with hard-to-treat or returning solid tumors, potentially slowing disease or shrinking tumors.
- What could go wrong
- This is an early-phase trial, so the combination may not work for all tumor types. Chemotherapy also carries risks like low blood counts, fatigue, and increased infection risk.
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 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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129 people
The number who actually took part.
- Start date
-
Jun 2009
- Finished
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Aug 2015
- 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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6 months to 20 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: * Histologically or cytologically confirmed neuroblastoma, brain tumor or other solid tumor (at diagnosis) * Relapsed or refractory tumors in which correct standard treatment approaches have failed * No more than 2 lines of prior chemotherapy * Measurable primary and/or metastatic disease on CT/MRI at least one bi-dimensionally measurable lesion. For patients with neuroblastoma, measurable disease will be defined by the modified International Neuroblastoma Staging System (Brodeur et al.1993) completed with MIBG scoring. * Age at inclusion: 6 months to ≤ 20 years * Lansky play score ≥ 70% or ECOG performance status ≤ 1 * Life expectancy ≥ 3 months * Adequate organ function: Adequate haematological function: haemoglobin ≥ 80 g/l, neutrophil count ≥ 1.0 x 109/L, platelet count ≥ 100 x 109/L; in case of bone marrow disease: neutrophils ≥ 0.5 x 109/l and platelets ≥ 75 x 109/l; Adequate renal function: normal creatinine related to patient's age: * 0 - 1 year: ≤ 40 µmol/L * 1 - 15 years: ≤ 65 µmol/L * 15 - 20 years: ≤ 110 µmol/L Adequate hepatic function: bilirubin ≤ 1.5 x ULN; AST and ALT ≤ 2.5 x ULN (AST, ALT ≤5xULN in case of liver metastases) * Wash-out of 4 weeks in case of prior chemotherapy, 6 weeks if treatment included nitrosoureas, 2 weeks in case of vincristine alone; 6 weeks in case of prior radiotherapy (except palliative radiotherapy on non measurable lesions). Patients must have recovered from the acute toxic effects of all prior therapy before enrolment into the study. * Patients previously treated with only one of the 2 drugs are eligible. * Able to comply with scheduled follow-up and with management of toxicity. * All patients with reproductive potential must practice an effective method of birth control while on study. Female patients aged \> 12 years must have a negative pregnancy test within 7 days before study treatment. * Written informed consent from patient, parents or legal guardian. Exclusion Criteria: * Concurrent administration of any other anti-tumour therapy. * Serious concomitant systemic disorder (for example, active infection including HIV or cardiac disease) that in the opinion of the investigator, would compromise the patient's ability to complete the study. * History of allergic reaction to the compounds or their solvents. * History of allergic reaction to Dacarbazine (DITC). * Galactosemia, Glucose-galactose malabsorption or lactase deficiency. * Pregnant or breast feeding young women. * Presence of symptomatic brain metastases in patients with solid non-CNS tumors.
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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
-
Institut Gustave Roussy
Villejuif, 94805, France
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