Topotecan IT + Maintenance Chemotherapy for Brain Cancer
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores a new treatment for very young children with specific aggressive brain tumors. It combines topotecan, a chemotherapy drug delivered directly into the spinal fluid (intrathecal chemotherapy), with ongoing maintenance therapy to assess feasibility and potential effectiveness. The trial targets children under 6 years old who have been newly diagnosed with certain central nervous system tumors, such as specific forms of medulloblastoma or atypical teratoid rhabdoid tumors. Participants should not have received prior cancer treatments beyond corticosteroids. As an Early Phase 1 trial, this research aims to understand how the treatment works in very young patients, offering a unique opportunity to be among the first to receive this novel approach.
Will I have to stop taking my current medications?
The trial requires that you do not take certain medications, like enzyme-inducing anticonvulsants and some antifungal drugs. It's best to discuss your current medications with the trial team to see if any need to be stopped or adjusted.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
Research has shown that administering topotecan directly into the spinal fluid is generally well-tolerated. In several studies, patients who received this treatment did not experience severe side effects. For example, one study found it effective without causing major problems, similar to other treatments administered in the same way. Another study also found this method safe for treating certain types of cancer in the brain and spinal cord.
Overall, evidence suggests that this approach is promising and has a manageable safety profile. However, as this is an early phase trial, not all possible side effects may be known yet. Participants should discuss any concerns with their healthcare provider.12345Why are researchers excited about this trial's treatments?
Researchers are excited about these treatments for brain cancer because they offer a comprehensive approach that targets the disease in multiple phases. Unlike standard treatments, which often focus on a single line of attack, this regimen uses a combination of intrathecal topotecan and personalized maintenance chemotherapy. Topotecan is delivered directly into the cerebrospinal fluid, potentially improving its effectiveness against brain tumors. Additionally, the use of autologous stem cell rescue during consolidation is a unique feature that could help patients tolerate higher doses of chemotherapy. This multifaceted strategy aims to address the cancer more thoroughly, offering hope for improved outcomes.
What evidence suggests that this trial's treatments could be effective for brain cancer?
Research has shown that injecting topotecan directly into the spinal fluid holds promise for treating certain brain and spinal cord cancers. In this trial, participants will receive monthly intrathecal topotecan as part of a comprehensive treatment regimen. Some studies have found that patients receiving this treatment experienced similar outcomes to those using other chemotherapy drugs, particularly in terms of how long their cancer remained stable. The treatment is generally well-tolerated, with most people not experiencing severe side effects. Additionally, patients in better health at the start of treatment tend to achieve better results. While the findings are hopeful, further research is needed to understand the treatment's effectiveness for brain cancer in young children.12346
Who Is on the Research Team?
Sylvia Cheng
Principal Investigator
BC Cancer Centre
Lucie Lafay-Cousin
Principal Investigator
Alberta Children's Hospital
Annie Huang
Principal Investigator
The Hospital for Sick Children
Are You a Good Fit for This Trial?
This trial is for children under 6 with high-risk embryonal brain tumors (HR-EBTs), including various specific types like ATRT and medulloblastoma. They must have a sample of the tumor tissue, an MRI of the brain and spine, a life expectancy over 8 weeks, and be able to perform daily activities at least half of what's expected for their age. Kids can't join if they have heart or kidney problems or trouble breathing.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Induction
Participants undergo three 21-day cycles of multiagent chemotherapy induction with intrathecal cytarabine and hydrocortisone, vinCRIStine, cyclophosphamide, CISplatin, and etoposide.
Consolidation
Participants receive three 28-day cycles of CARBOplatin and thiotepa followed by autologous stem cell rescue.
Maintenance
Participants receive risk-stratified maintenance chemotherapy for 48-54 weeks, including monthly intrathecal topotecan and either 'Maintenance A' or 'Maintenance B' regimens.
Follow-up
Participants are monitored for progression-free survival and overall survival every 3 months for 24 months after completion of treatment.
What Are the Treatments Tested in This Trial?
Interventions
- Intrathecal Chemotherapy
- Maintenance Chemotherapy
- Topotecan
Trial Overview
The study tests adding intrathecal chemotherapy (directly into the spinal fluid) and ongoing maintenance therapy after intense chemo in young patients with HR-EBTs. Drugs include carboplatin, thiotepa, isotretinoin, cyclophosphamide, cisplatin, mesna, vincristine, filgrastim etoposide phosphate cytarabine IT hydrocortisone celecoxib etoposide topotecan IT tamoxifen temozolomide.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will undergo a comprehensive treatment regimen beginning with three 21-day cycles of Induction chemotherapy including intrathecal (IT) cytarabine with hydrocortisone, cyclophosphamide, etoposide, vinCRIStine, and cisplatin. Peripheral blood stem cells will be collected during this phase for later use. Patients who achieve complete response (CR) proceed directly to Consolidation; those who do not may undergo second-look surgery or national tumor board review. Consolidation consists of three 28-day cycles of CARBOplatin and thiotepa followed by autologous stem cell rescue. Patients then proceed to up to 48-54 weeks of Maintenance chemotherapy based on risk stratification. Low-risk patients receive monthly IT topotecan and a 28-day metronomic regimen including tamoxifen and ISOtretinoin. High-risk patients receive monthly IT topotecan and a more intensive regimen every 9 weeks including ISOtretinoin, celecoxib, etoposide, cyclophosphamide, and temozolomide.
Find a Clinic Near You
Who Is Running the Clinical Trial?
C17 Council
Lead Sponsor
Citations
A multicenter phase II trial of intrathecal topotecan in ...
IVent topotecan is well tolerated, and its use results in CSF clearance rates, progression, and survival outcomes similar to those of other IVent agents.
LPTO-09. INTRATHECAL TOPOTECAN FOR ...
CONCLUSIONS: IT topotecan has a modest side effect profile. Patients with higher functional status at diagnosis had significantly better ...
A review on intrathecal administration of medications for ...
Researchers treated two patients with recurrent or progressive CNS cancer with LMD using IT topotecan (0.2 mg/day) for 7 days in order to increase the exposure ...
Phase 1 Intrathecal Topotecan for Neoplastic Meningitis
Consolidation (6 weeks): Patients will receive IT topotecan for 5 consecutive days during weeks 1 and 4. The Consolidation period is 6 weeks in duration.
Case studies of patients receiving intraventricular ...
The use of IVt topotecan in the treatment of NM results in progression-free survival outcomes similar to other IVt chemotherapies, while being ...
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cdn.amegroups.cn/journals/pbpc/files/journals/1/articles/87417/public/87417-PB1-3712-R1.pdfIntrathecal topotecan with systemic checkpoint inhibitor ...
Two patients with leptomeningeal metastases (LM) from their gastroesophageal malignancy have demonstrated excellent response to combination ...
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