Introduction

This family protocol summary is about the Children's Oncology Group study ACNS1422. It tells who is eligible and gives basic information about the study. More details about the study are in the consent form and protocol. You can get these from your oncologist.

ACNS1422 is a Phase 2 clinical trial. A trial is another word for a study. This study (clinical trial) is a therapeutic clinical trial. That means it is done to learn about treatment -- its safety and how well it works. The purpose of a Phase 2 trial is to learn if a new treatment works in treating a type of cancer and how it affects the body.

Patients on a Phase 2 trial are checked for side effects of the treatment and to see whether the cancer shrinks, goes away or comes back.

In most clinical trials, we will not know which treatment is better until all of the children taking part in the trial have completed treatment and have been getting checked for several years. If one of the treatments is found to be better or safer than the others while the trial is still going on, the trial will be stopped. If a treatment is found to be worse while the trial is still going on, the trial will be stopped. All children still in the trial will be given the treatment with the best results when possible.

It is important to understand that participating in a long-term follow-up study is entirely voluntary. The decision about whether or not to participate will not affect the care provided by the healthcare team in any way. You can find additional information about participation in non-therapeutic studies at www.childrensoncologygroup.org. Always discuss any questions that you may have with your health care team.

Study Number

ACNS1422

Official Title

A Phase 2 Study of Reduced Therapy for Newly Diagnosed Average-Risk WNT-Driven Medulloblastoma Patients

Trial Opening Date

ACNS1422 opened on 10/02/2017

General Patient Eligibility

• Age: 3 years of age or older
• Diagnosis: Newly diagnosed, average risk Medulloblastoma, in the WNT subgroup

Please consult your doctor to determine whether you or your child may participate in this study.

General Background and Study Goal

WNT is one of the four separate subgroups of medulloblastoma defined by a unique molecular profile. Patients with this tumor have an excellent prognosis with standard therapy. This study is looking at the possibility of giving a lower dose of radiation to the head and spine and lowering the amount of chemotherapy as compared to standard therapy to reduce long-term toxicity, without risking the tumor coming back.

The goals of this study are to:

• See if children ages 3 years and older with WNT-driven average risk medulloblastoma will continue with excellent survival rates with less radiation and less chemotherapy.
• Better understand the biology of WNT subtype Medulloblastoma.
• Closely examine the brain function as it relates to thinking and learning in children with WNT subtype Medulloblastoma after they receive treatment on this study.

Summary of the Treatment

Patients with medulloblastoma that has not spread (non-metastatic) and who are at least 3 years old will be enrolled on the APEC14B1 study. APEC14B1, or the Project Every Child study, runs extra tests on your blood and tumor tissue to determine if the tumor is a WNT-subtype medulloblastoma. About a teaspoon (5 mL) of blood will be taken at the same time blood is drawn for standard blood tests. Left over tumor after diagnostic tests are completed will also be studied. This must be done within 15 days of surgery.

If it is determined that a patient has WNT-driven medulloblastoma, they will be enrolled on ACNS1422. Therapy consists of:

• 6 weeks of radiation therapy, to begin 4-5 weeks after surgery
  • 18 Gy (unit of radiation) craniospinal (head and spine) + boost to the tumor area (as compared to 24 Gy given in standard therapy)
• No vincristine during radiation (as compared to vincristine given weekly for 6 weeks during standard therapy)
• 7 cycles of maintenance chemotherapy (as compared to 9 cycles in standard therapy), alternating between cycle A and B: ABABABA). Maintenance chemotherapy will begin 4 weeks after radiation therapy is complete.
  • Cycle A (42 Days)
    • Cisplatin intravenous (IV) on Day 1
    • Lomustine (CCNU) by mouth on Day 1
    • Vincristine IV on Days 1, 8, and 15
  • Cycle B (28 Days)
    • Cyclophosphamide IV on Days 1 and 2
    • MESNA IV on Days 1 and 2
    • Vincristine IV on Days 1 and 8

Risks and Side Effects

Chemotherapy can cause side effects during and after treatment. All risks and side effects will be explained in detail by your healthcare team during the consent process. They can answer any questions that you may have about giving permission for your child to be in the clinical trial or other aspects of care. Please refer to the consent form for a detailed explanation of the side effects associated with the treatment on this study. You can choose to come off of the study at any time. Discuss this with your healthcare team.

Contact Information

You or your child's oncologist and nurses are the best source for further information.

Study Chair
Nicholas Gottardo, MD
Perth Children’s Hospital, Perth, Australia

FAMILY PROTOCOL SUMMARY REVIEW/APPROVALS