PNOC044: Liothyronine (L-T3) in combination with bevacizumab, irinotecan and temozolomide (BIT) for progressive or relapsed medulloblastoma (Cohort 1) or as monotherapy for medulloblastoma with minimal residual disease (Cohort 2)
PNOC044
What is the goal of the study?
This is a Phase 1/Phase 2 study assessing liothyronine (L-T3) monotherapy and in combination with standard chemotherapy (bevacizumab, irinotecan and temozolomide (BIT)) in children and young adults with medulloblastoma that is relapsed or progressive after standard upfront therapy. The Phase 1 portion will establish recommended Phase 2 dose (RP2D) of L-T3 in combination with BIT. The RP2D of L-T3 will be used in the Phase 2 portion to assess its efficacy when given in combination with BIT and its efficacy as monotherapy. The efficacy of the L-T3 RP2D in combination with BIT will be evaluated in participants with imaging evidence of relapsed/progressive medulloblastoma (cohort 1). The efficacy of L-T3 monotherapy will be assessed in children and young adults with medulloblastoma with cerebrospinal fluid (CSF) cell-free DNA (cfDNA) positivity without radiographic evidence of disease progression/recurrence following standard upfront therapy (cohort 2).
Who can participate in the study?
Please contact the study team listed below to learn more.