Maintenance Chemotherapy vs Observation for Ependymoma

Recruiting in Palo Alto (17 mi)

+212 other locations

Overseen byAmy A Smith

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Children's Oncology Group

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

The primary aim of this randomized phase III trial was to study whether the addition of maintenance chemotherapy delivered after surgical resection and focal radiation would be better than surgery and focal radiation alone. The trial also studied if patients who received induction chemotherapy and then either achieved a complete response or went on to have a complete resection would also benefit from maintenance chemotherapy. Children ages 1-21 years with newly diagnosed intracranial ependymoma were included. There were 2 arms that were not randomized. One arm studied patients with Grade II tumors located in the supratentorial compartment that were completely resected. One arm studied patients with residual tumor and those patients all received maintenance chemotherapy after focal radiation. Chemotherapy drugs, such as vincristine sulfate, carboplatin, cyclophosphamide, etoposide, and cisplatin, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Radiation therapy uses high-energy x-rays to kill tumor cells. Specialized radiation therapy that delivers a high dose of radiation directly to the tumor may kill more tumor cells and cause less damage to normal tissue. Giving chemotherapy in combination with radiation therapy may kill more tumor cells and allow doctors to save the part of the body where the cancer started.

Research Team

Amy A Smith

Principal Investigator

Children's Oncology Group

Eligibility Criteria

This trial is for children and young adults aged 1-21 with newly diagnosed brain ependymoma, who've had surgery but no other treatment. It's not for those with spinal cord ependymoma or certain other types, pregnant or breastfeeding individuals, or patients with metastatic disease.

Inclusion Criteria

My child has ependymoma but can still participate in the study despite any neurological issues.

I have been newly diagnosed with a specific type of brain tumor called ependymoma.

REGULATORY: All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met

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Exclusion Criteria

I have only had surgery or used corticosteroids for my condition.

I do not have cancer spread beyond the primary site as confirmed by MRIs and CSF tests.

Pregnant female patients are not eligible for this study

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Treatment Details

Interventions

3-Dimensional Conformal Radiation Therapy (Radiation)
Carboplatin (Alkylating agents)
Cisplatin (Platinum-containing compounds)
Clinical Observation (Other)
Cyclophosphamide (Alkylating agents)
Etoposide (Topoisomerase II inhibitors)
Laboratory Biomarker Analysis (Other)
Vincristine (Vinca alkaloids)

Trial OverviewThe study tests if maintenance chemotherapy after surgery and radiation helps more than just surgery and radiation alone in treating brain ependymoma. Some will receive combination chemotherapy drugs plus specialized radiation therapy aimed at killing tumor cells while sparing healthy tissue.

Participant Groups

4Treatment groups

Experimental Treatment

Active Control

Group I: Arm II (radiotherapy, chemotherapy)Experimental Treatment8 Interventions

Patients with supratentorial ependymoma (Grade II without GTR1 or Grade III) or any infratentorial ependymoma who have undergone gross or near total resection (GTR or NTR) or achieved CR either after first or second resection or after post-operative induction chemotherapy are randomized to undergo conformal radiotherapy over 6-7 weeks followed by maintenance chemotherapy. Maintenance chemotherapy comprised of vincristine IV on days 1, 8, and 15 of cycles 1-3 only, etoposide IV over 60-120 minutes on days 1-3, cisplatin IV over 1-8 hours on day 1, and cyclophosphamide IV over 30-60 minutes on days 2-3. Treatment repeats every 21 days for 4 courses in the absence of disease progression or unacceptable toxicity. Patients without GTR or NTR at enrollment require induction chemotherapy (see Arm I) and possibly second surgery before randomization.

Group II: Arm I (chemotherapy, observation)Experimental Treatment7 Interventions

Patients with classic histology(WHO Grade II) supratentorial ependymoma who have undergone microscopic gross total resection (GTR1) or achieved CR either after first or second resection or after post-operative induction chemotherapy are assigned to observation. For patients without GTR1, induction chemotherapy is comprised of vincristine intravenously (IV) over 1 minute on days 1 and 8 of cycles 1 and 2, carboplatin IV over 15-60 minutes on day 1 of cycles 1 and 2, and cyclophosphamide IV over 30-60 minutes on days 1-2 of cycle 1 only. Patients also receive etoposide IV over 60-120 minutes on days 1-3 of cycle 2 only. Cycle 1 continues for 3 weeks and cycle 2 continues for 4 weeks in the absence of disease progression or unacceptable toxicity.

Group III: ARM IV (radiotherapy, chemotherapy)Active Control8 Interventions

Patients with subtotal resection after induction chemotherapy (see Arm I) and second surgery are nonrandomly assigned to Arm II treatment.

Group IV: Arm III (radiotherapy, observation)Active Control3 Interventions

Patients with supratentorial ependymoma (Grade II without GTR1 or ST Grade III) or any infratentorial ependymoma (Grade II or III) who have undergone gross or near total resection or achieved CR either after first or second resection or after post-operative induction chemotherapy are randomized to undergo conformal radiotherapy over 6-7 weeks and then undergo observation. Patients without GTR or NTR at enrollment require induction chemotherapy (see Arm I) and possibly second surgery before randomization.

3-Dimensional Conformal Radiation Therapy is already approved in Canada, Japan, China, Switzerland for the following indications: