Newly Diagnosed Medulloblastoma
Conditions
Brief summary
Progression-Free Survival (PFS) according to RAPNO assessed by central reviewer of a personalized intensity-modulated therapy (experimental arm; sonidegib) compared to standard therapy in SHH-activated patients in post-pubertal patients with newly diagnosed standard risk medulloblastoma.
Detailed description
Progression-Free Survival (PFS) by central reviewer in WNT and Group 3 & Group 4 subgroups, Progression-Free Survival (PFS) by local investigator, Overall survival (OS), Frequencies and percentages of worst adverse events (AEs) or laboratory event; grades according to CTCAE v.5 (with neurological, kidney, auditory, endocrine and radiotherapy associated as AE of special interest), Patient reported outcomes: • Health-related Quality of life (HRQol): EORTC QLQ-C30 and BN20, social functioning as scale of special interest • Survivorship outcomes (five items from EORTC QLQ-SURV111) • Neurocognitive function (NCF): Hopkins Verbal Learning Test, Controlled Oral Word Association Trail Making Test Part A, Trail Making Test Part B, cerebellar cognitive affective/Schmahmann syndrome scale.
Interventions
Sponsors
European Organisation For Research And Treatment Of Cancer
Eligibility
Sex/Gender
All
Age
0 Years to No maximum
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Progression-Free Survival (PFS) according to RAPNO assessed by central reviewer of a personalized intensity-modulated therapy (experimental arm; sonidegib) compared to standard therapy in SHH-activated patients in post-pubertal patients with newly diagnosed standard risk medulloblastoma. | — |
Secondary
| Measure | Time frame |
|---|---|
| Progression-Free Survival (PFS) by central reviewer in WNT and Group 3 & Group 4 subgroups, Progression-Free Survival (PFS) by local investigator, Overall survival (OS), Frequencies and percentages of worst adverse events (AEs) or laboratory event; grades according to CTCAE v.5 (with neurological, kidney, auditory, endocrine and radiotherapy associated as AE of special interest), Patient reported outcomes: • Health-related Quality of life (HRQol): EORTC QLQ-C30 and BN20, social functioning as scale of special interest • Survivorship outcomes (five items from EORTC QLQ-SURV111) • Neurocognitive function (NCF): Hopkins Verbal Learning Test, Controlled Oral Word Association Trail Making Test Part A, Trail Making Test Part B, cerebellar cognitive affective/Schmahmann syndrome scale. | — |
Countries
Germany
Outcome results
None listed