A Pilot Study Investigating Upfront Adaptive Immunotherapy Approach in Children, Adolescent and Young Adult (CAYA) Patients With Replication-Repair Deficient (RRD) High-Grade Gliomas (HGG) (NCT06519682)

🧪 Interventions in This Study

• Nivolumab (DRUG)

Check every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).

1. 1.1 Age: Patients must be ≥12 months and ≤25 years of age at the time of signing informed consent/assent.
2. 1.2 Diagnosis: Patients must have a histologically confirmed diagnosis of glioblastoma.
3. 1.3 Proof of RRD: By tumor immunohistochemistry showing functional loss of mismatch repair gene expression (MLH1, MSH2, MSH6, PMS2), or based on prior germline testing confirming congenital mismatch repair deficiency (CMMRD) or Lynch syndrome. To be done locally. Results have to be available within four weeks of last surgery.
4. 1.4 Tumor Tissue Specimen: Provide a tumor tissue specimen for molecular profiling, including TMB analysis. Any tumor sequencing data if available at time of enrolment will be recorded for relevant pathogenic variants in the mismatch repair and polymerase-proofreading genes to suggest RRD. A specimen from the time of relapse/ progression while on the study is required as well, when applicable.
5. 1.5 Favorable immune markers: High PD1 and CD8 positivity as detailed in the lab manual. To be done locally. Results have to be available within 4 weeks of last surgery.
6. 1.6 Surgical and disease status: Patients should have had Gross total resection (GTR)/Near Total Resection (NTR) as confirmed by the post-surgery scan. Patients are allowed a second look surgery to achieve NTR/GTR provided no tumor directed systemic or radiation therapy has been administered before this second surgery. Patients should be able to start therapy within 4 weeks of last surgery.
7. 1.7 Allowable Prior Therapy: 3.1.7.1 Patients must have recovered from the acute toxic effects of all prior anti-cancer therapies (with the exception of alopecia and lymphopenia). Previous treatment with nivolumab and/or other anti- PD-1/PD-L1 inhibitors for other prior tumors (other than high-grade glioma) will be permitted.
8. 1.7.2 Prior Therapy: No prior therapy except surgery will be permitted for high grade glioma. If the patient was previously diagnosed and treated for another tumor (other than high grade glioma), the patients must have completed that treatment and have no active disease in order to be enrolled in this trial The following time periods apply for prior therapy for other tumors:
9. Cytotoxic chemotherapy: At least 21 days prior to initiation of protocol therapy from the last dose of cytotoxic or myelosuppressive chemotherapy; at least 42 days if prior nitrosourea (such as lomustine, CCNU).
10. Hematopoietic growth factors: At least 7 days prior to initiation of protocol therapy from the last dose of short-acting growth factor; at least 14 days for long-acting.
11. Anti-cancer agents not known to be myelosuppressive: At least 7 days prior to initiation of protocol therapy from the last dose.
12. Interleukins, interferons, and cytokines (other than hematopoietic growth factors): At least 21 days prior to initiation of protocol therapy from the last dose.

🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.

📍 All Participating Trial Locations (1 Sites)

• Hospital for Sick Children — Toronto, Ontario

📞 Contact the Study Coordinator

Phone: (613) 737-7600

Email: Nthacker@cheo.on.ca

Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.

💰 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.
• 💵 Compensation indicated: Patients may receive up to a total of approximately 2 years of treatment (up to 24 cycles).

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• Lutathera for Treatment of Recurrent or Progressive High-Grade CNS Tumors — Aurora, Colorado
• MRI in High-Grade Glioma Patients Undergoing Chemoradiation — Winston-Salem, North Carolina
• A Pilot Study of Larotrectinib for Newly-Diagnosed High-Grade Glioma With NTRK Fusion — Aurora, Colorado
• Lorlatinib for Newly-Diagnosed High-Grade Glioma With ROS or ALK Fusion — Aurora, Colorado

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💡 How to Participate

1. Check the checklist above to see if you may qualify.
2. Contact the study coordinator using the phone/email above.
3. Ask about the visit schedule, what is covered at no cost, and any travel support.
4. Confirm with your own doctor before making a decision.