A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma (NCT01878617)
🩺 Plain-English Summary (8th-Grade Level)
Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk). Recent studies have shown that medulloblastoma is made up of distinct molecular subgroups which respond differently to treatment. This suggests that clinical risk alone is not adequate to identify actual risk of recurrence. In order to address this, we will stratify medulloblastoma treatment in this phase II clinical trial based on both clinical risk (low, standard, intermediate, or high risk) and molecular subtype (WNT, SHH, or Non-WNT Non-SHH). This stratified clinical and molecular treatment approach will be used to evaluate the following: * To find out if participants with low-risk WNT tumors can be treated with a lower dose of radiation to the brain and spine, and a lower dose of the chemotherapy drug cyclophosphamide while still achieving the same survival rate as past St. Jude studies with fewer side effects. * To find out if adding targeted chemotherapy after standard chemotherapy will benefit participants with SHH positive tumors. * To find out if adding new chemotherapy agents to the standard chemotherapy will improve the outcome for intermediate and high risk Non-WNT Non-SHH tumors. * To define the cure rate for standard risk Non-WNT Non-SHH tumors treated with reduced dose cyclophosphamide and compare this to participants from the past St. Jude study. All participants on this study will have surgery to remove as much of the primary tumor as safely possible, radiation therapy, and chemotherapy. The amount of radiation therapy and type of chemotherapy received will be determined by the participant's treatment stratum. Treatment stratum assignment will be based on the tumor's molecular subgroup assignment and clinical risk. The participant will be assigned to one of three medulloblastoma subgroups determined by analysis of the tumor tissue for tumor biomarkers: * WNT (Strata W): positive for WNT biomarkers * SHH (Strata S): positive for SHH biomarkers * Non-WNT Non-SHH, Failed, or Indeterminate (Strata N): negative for WNT and SHH biomarkers or results are indeterminable Participants will then be assigned to a clinical risk group (low, standard, intermediate, or high) based on assessment of: * How much tumor is left after surgery * If the cancer has spread to other sites outside the brain \[i.e., to the spinal cord or within the fluid surrounding the spinal cord, called cerebrospinal fluid (CSF)\] * The appearance of the tumor cells under the microscope * Whether or not there are chromosomal abnormalities in the tumor, and if present, what type (also called cytogenetics analysis)
Who can join: Review the eligibility criteria below.
Location: Lucille Packard Children's Hospital at Stanford University Medical Center — Palo Alto, California
Age range: 3 Years to 39 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (23 Sites)
- Lucille Packard Children's Hospital at Stanford University Medical Center — Palo Alto, California
- Rady Children's Hospital — San Diego, California
- Yale University — New Haven, Connecticut
- Children's National Medical Center — Washington D.C., District of Columbia
- University of Florida — Gainesville, Florida
- Arnold Palmer Hospital for Children — Orlando, Florida
- Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota
- Duke Children's Hospital and Health Center — Durham, North Carolina
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Medical University of South Carolina — Charleston, South Carolina
- St. Jude Children's Research Hospital — Memphis, Tennessee
- University of Texas Southwestern Medical Center — Dallas, Texas
- Cook Children's Medical Center — Fort Worth, Texas
- Texas Children's Cancer Center — Houston, Texas
- Sydney Children's Hospital — Randwick, New South Wales
- Children's Hospital at Westmead — Westmead, New South Wales
- Queensland Children's Hospital — Brisbane, Queensland
- Royal Children's Hospital, Melbourne — Melbourne, Victoria
- Perth Children's Hospital — Perth, Western Australia
- Alberta Children's Hospital — Calgary, Alberta
- The Hospital for Sick Children — Toronto, Ontario
- Centre Hospitalier Universitaire Sainte-Justine — Montreal, Quebec
- Starship Children's Hospital — Auckland,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Medulloblastoma
- Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma — Birmingham, Alabama
- A Pilot Study of SurVaxM in Children Progressive or Relapsed Medulloblastoma, High Grade Glioma, Ependymoma and Newly Diagnosed Diffuse Intrinsic Pontine Glioma — Los Angeles, California
- HeadStart4: Newly Diagnosed Children (<10 y/o) With Medulloblastoma and Other CNS Embryonal Tumors — Birmingham, Alabama
- Comparison of Neurocognitive Outcome in Two Standard Regimen for Treatment of Low-risk Medulloblastoma — Birmingham, Alabama
- Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease — San Francisco, California
Explore by condition: All Medulloblastoma clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.