Standard Regimen Comparison for Medulloblastoma

Not yet recruiting at 1 trial location
KT
Overseen ByKelsey Troyer, PhD
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Nationwide Children's Hospital
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial compares two chemotherapy treatments to determine which better preserves brain function in children diagnosed with medulloblastoma, a specific type of brain cancer. It evaluates children's outcomes 2.5 years after diagnosis when treated with either the "Head Start 4" or HIT-SKK treatment plan, which includes Bridging Chemotherapy, Induction Cycles A1-A3, Induction Cycles A4-5, HIT-SKK Chemotherapy Cycles B1-3, and Modified HIT-SKK Cycle B4-5. Children under 5 years old diagnosed with this non-metastatic form of medulloblastoma who meet specific medical criteria are suitable candidates for the trial. Participants must be able to complete follow-up brain function tests and have family support for regular check-ups. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking treatment advancements.

Do I need to stop my current medications to join the trial?

The trial information does not specify if you need to stop taking your current medications. However, you cannot use live vaccines during the study, and there should be no medical contraindications to chemotherapy.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that the "Head Start 4" chemotherapy treatment is effective for young children with medulloblastoma, a type of brain cancer. Most patients responded well, though it can cause side effects such as nausea or tiredness, like all chemotherapy.

Past studies have also shown good results for the HIT-SKK chemotherapy, especially in young children with medulloblastoma that hasn't spread. This treatment combines regular chemotherapy with a method that delivers medicine directly into the brain fluid. Many patients have done well for at least five years after treatment. While it can have side effects, it has been used safely in previous studies.

This trial compares both treatments to determine which might better support brain function and thinking skills in children with a specific type of medulloblastoma.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about the treatments in the Standard Regimen Comparison for Medulloblastoma trial because they offer innovative approaches to chemotherapy. Unlike the traditional regimens, Arm A involves the "Head Start" 4 approach, which combines multiple induction chemotherapy cycles with a high-dose cycle, potentially enhancing the overall effectiveness and reducing long-term side effects. Arm B, on the other hand, builds on previous successful HIT-SKK studies, aiming to refine and optimize chemotherapy delivery over several cycles. These methods seek to improve treatment outcomes and quality of life for patients with medulloblastoma by offering more tailored and potentially less toxic options compared to standard treatments like craniospinal irradiation and conventional chemotherapy.

What evidence suggests that this trial's treatments could be effective for medulloblastoma?

Research shows that the "Head Start 4" chemotherapy method, a treatment arm in this trial, yields promising results for children with medulloblastoma, particularly in improving survival rates and quality of life. Previous studies with similar treatments have demonstrated good outcomes for young patients. The HIT-SKK chemotherapy plan, another treatment arm in this trial, achieved a 93% five-year progression-free survival (PFS) rate for low-risk patients, indicating that many patients did not experience cancer progression for at least five years post-treatment. Both treatment options in this trial have strong evidence supporting their effectiveness in treating medulloblastoma in children.13467

Who Is on the Research Team?

MF

Maryam Fouladi, MD

Principal Investigator

Nationwide Children's Hospital

GD

Girish Dhall, MD

Principal Investigator

Children's Hospital of Alabama

Are You a Good Fit for This Trial?

This trial is for children under 5 with a specific brain tumor called SHH-activated, TP53-wt medulloblastoma without MYC amplification. They must not have had previous cancer treatments other than surgery, no severe organ dysfunction or infections, and be able to undergo MRI and neuropsychological tests. Families must commit to follow-up studies.

Inclusion Criteria

My family and I can attend follow-up mental health checks.
Ability of parents/legal representatives to understand the patient information and to personally sign and date the informed consent to participate in screening procedures
I and my legal guardian are willing to participate in the study.
See 23 more

Exclusion Criteria

I have a severe developmental delay that prevents me from completing certain assessments.
Patients cannot undergo MRI
My cancer is MYC amplified.
See 17 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction Chemotherapy

Participants receive 3 to 5 cycles of induction chemotherapy as part of the 'Head Start' 4 or HIT-SKK regimen

12-20 weeks

Consolidation Chemotherapy

Participants in Arm A receive high-dose chemotherapy as part of the 'Head Start' 4 regimen

4-6 weeks

Follow-up

Participants are monitored for neurocognitive outcomes, survival, and other health impacts 2.5 years after diagnosis

2.5 years

What Are the Treatments Tested in This Trial?

Interventions

  • Bridging Chemotherapy
  • Consolidation Cycle A6
  • HIT-SKK Chemotherapy Cycles B1-3
  • Induction Cycles A1-A3
  • Induction Cycles A4-5
  • Modified HIT-SKK Cycle B4-5

Trial Overview

The study compares two chemotherapy regimens (Head Start 4 vs HIT-SKK) in treating low-risk medulloblastoma to see which one leads to better neurocognitive outcomes after 2.5 years. It involves various cycles of induction and consolidation chemotherapies.

How Is the Trial Designed?

2

Treatment groups

Experimental Treatment

Group I: Arm B: HIT-SKKExperimental Treatment3 Interventions
Group II: Arm A: "Head Start" 4Experimental Treatment4 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Nationwide Children's Hospital

Lead Sponsor

Trials
354
Recruited
5,228,000+

German Society of Paediatric Oncology and Hematology (GPOH gGmbH)

Collaborator

Children's of Alabama

Collaborator

Trials
10
Recruited
3,000+

Citations

Comparison of Neurocognitive Outcome in Two Standard ...

This is a trial to compare neurocognitive outcomes in the intent-to-treat population 2.5 years after diagnosis between patients with newly ...

Comparison of Neurocognitive Outcome in Two Standard ...

This regimen combines systemic chemotherapy with intraventricular MTX, leading to 93% 5-year PFS in low-risk patients. Both treatment regimens ...

Medulloblastoma: Current Perspectives and Recent Advances

3 cycles of HIT-SKK chemotherapy with intraventricular MTX → 2 cycles of modified HIT-SKK chemotherapy, 5-yr PFS 93% for DN or MBEN patients, 5-yr PFS 37 ...

Medulloblastoma

Patients with low risk iMB will receive 5 cycles of HIT SKK chemotherapy as per HIT2000. Systemic chemotherapy should commence within 28 days of surgery.

Review of the impact of molecular analysis on the therapy ...

This review summarizes recent advances in the molecular characterization of medulloblastoma and evaluates subgroup directed treatment.

Treatment of young children with localized medulloblastoma ...

This study was designed to confirm the previously observed favorable survival rates and prognostic factors in young children with nonmetastatic ...

Consensus treatment recommendations from SIOP-Europe ...

Patients with low risk iMB will receive 3 cycles (5 if residual disease) of HIT SKK chemotherapy as per HIT2000. Systemic chemotherapy should commence at the ...