NCT04402073

Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma

Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma (PersoMed-I)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: European Organisation for Research and Treatment of Cancer - EORTC · Network
• Sex: All
• Age: 15 Years
• Healthy volunteers: Not accepted

Summary

Medulloblastoma is a rare brain malignancy, mainly affecting children. Treatment of this rapidly growing tumor begins with maximal surgical removal plus radiation and chemotherapy. Treatment toxicity is high. Post-pubertal and pediatric medulloblastomas are biologically and prognostically different, which mandates age-adapted treatment strategies. Patients after puberty bear an intermediate to high prognostic risk. This means that a large number of these patients, are faced with death and/or disability (mainly neurocognitive). Therefore, the scientific and medical need is high. One of the genetic subgroups of medulloblastoma, the SHH-subgroup (Sonic HedgeHog- subgroup), is highly overrepresented in medulloblastoma patients after puberty. This subgroup can be treated with a targeted therapy. The investigators will therefore randomize patients and treat SHH-subgroup patients with sonidegib and a reduction of radiotherapy dose in the experimental arm of the trial. The hypothesis that this personalized risk-adapted therapy will improve outcomes in view of increased efficacy and decreased toxicity.

Conditions

• Medulloblastoma

Interventions

Type	Name	Description
DRUG	Sonidegib	Sonidegib is a selective smoothened inhibitor that inhibits the sonic hedgehog-signaling pathway. It is used in patients with locally advanced basal cell carcinoma (BCC) that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy.
DRUG	Cisplatin	Cisplatin (CIS) is a platinum derivate used in the treatment in several epithelial tumours. The application route is through the veins.
DRUG	Lomustine	Lomustine is a nitrosourea used in the treatment of brain tumours and Hodgkin's disease. The application route is oral.
DRUG	Vincristine	Vincristine sulfate (VCR) is an inhibitor of microtubule formation in the mitotic spindle, resulting in an arrest of dividing cells at the metaphase stage. Vincristine sulfate is indicated in acute leukemia, Hodgkin's disease, non-Hodgkin's lymphomas, rhabdomyosarcoma, neuroblastoma, and Wilms' tumor. The application route is through the vein.
RADIATION	radiotherapy	Radiotherapy to the cranio-spinal axis of 35.2 Gy in 22 daily fractions of 1.6 Gy, followed by an additional boost to the tumour site of 19.8 Gy in 11 daily fractions of 1.8 Gy, summing up to a total dose of 55.0 Gy in 33 daily fractions of 1.6/1.8 Gy.

Timeline

• Start date: 2022-11-11
• Primary completion: 2025-06-16
• Completion: 2025-09-10
• First posted: 2020-05-26
• Last updated: 2025-10-23

Locations

50 sites across 8 countries: Australia, Austria, France, Germany, Italy, Netherlands, Spain, Switzerland

Regulatory

• FDA-regulated drug study