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)
Sabine Mueller, MD, PhD
Summary
This is a Phase 1/Phase 2 study assessing liothyronine (L-T3) immunotherapy 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.
Description
OUTLINE: This is an intra-patient dose escalation (Phase 1 (Cohort 1)) followed by a Phase 2 (Cohort 2) study. Cohort 1: Children and young adults with relapsed or progressive disease following standard upfront therapy (including craniospinal radiation or high dose chemotherapy with autologous stem cell rescue). evaluating the safety and efficacy of combination L-T3 with BIT. Cohort 2: Children and young adults with medulloblastoma and CSF cell-free deoxyribonucleic acid (cf-DNA) positivity without radiographic disease progression/recurrence following standard upfront therapy. This is a Phase…
Eligibility
- Age range
- 1–25 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Phase 1 and Phase 2, Cohort 1: Participants must have histologically confirmed medulloblastoma that is relapsed/progressive following standard upfront therapy. Tissue confirmation of medulloblastoma diagnosis is required at diagnosis and not required at the time of relapse for entry into the study. 2. Phase 2, Cohort 2: Participants must have cerebrospinal fluid (CSF) with cell-free deoxyribonucleic acid (cf-DNA) + assessed in a Chemiluminescent immunoassay (CLIA)-certified or protocol-approved laboratory. After entry into the study, another CSF sample will be colle…
Interventions
- DrugLiothyronine (L-T3)
Given orally (PO)
- DrugBevacizumab
Given IV
- DrugIrinotecan
Given IV
- DrugTemozolomide (TMZ)
Given PO
Location
- University of California, San FranciscoSan Francisco, California