Ignite Creation Date:
2026-03-26 @ 3:16 PM
Ignite Modification Date:
2026-03-30 @ 1:32 AM
Study NCT ID:
NCT07346157
Status:
NOT_YET_RECRUITING
Last Update Posted:
2026-01-16
First Post:
2026-01-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease
Sponsor:
Sabine Mueller, MD, PhD
Organization:
Study Overview
Official Title:
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)
Status:
NOT_YET_RECRUITING
Status Verified Date:
2026-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
PNOC044
Brief 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.
Detailed 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 2 study to evaluate clearance of Circulating free DNA (cfDNA) positive disease in CSF in response to L-T3 monotherapy at the RP2D (established as part of Cohort 1; Phase 1) as single agent.
Participants may continue therapy for up to 12 cycles if there is no evidence of unacceptable toxicity, disease progression, or withdrawal of consent. For participants that are benefiting from therapy, they may continue L-T3 monotherapy for one additional year (24 cycles total therapy). Treatment beyond that specified in the protocol should be discussed with the study chairs. Duration of Follow up Participants will enter follow up after the 30-day toxicity period.
Follow-up procedures are to be captured under the Pediatric NeuroOncology Consortium (PNOC) COMP protocol with the exception of protocol defined follow up procedures. Participants will be followed under the PNOC COMP protocol until death or withdrawal from study.
The study will complete enrollment within 5 years from the time the study opens to accrual (including 3 years for enrollment and 2 years of long term follow up).
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 2 study to evaluate clearance of Circulating free DNA (cfDNA) positive disease in CSF in response to L-T3 monotherapy at the RP2D (established as part of Cohort 1; Phase 1) as single agent.
Participants may continue therapy for up to 12 cycles if there is no evidence of unacceptable toxicity, disease progression, or withdrawal of consent. For participants that are benefiting from therapy, they may continue L-T3 monotherapy for one additional year (24 cycles total therapy). Treatment beyond that specified in the protocol should be discussed with the study chairs. Duration of Follow up Participants will enter follow up after the 30-day toxicity period.
Follow-up procedures are to be captured under the Pediatric NeuroOncology Consortium (PNOC) COMP protocol with the exception of protocol defined follow up procedures. Participants will be followed under the PNOC COMP protocol until death or withdrawal from study.
The study will complete enrollment within 5 years from the time the study opens to accrual (including 3 years for enrollment and 2 years of long term follow up).
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| NCI-2025-08554 | REGISTRY | NCI Clinical Trials Reporting Program (CTRP) | View |