Liothyronine in Combination With BIT Regimen for Medulloblastoma With or Without Minimal Residual Disease

Conditions

• Medulloblastoma
• Medulloblastoma Recurrent
• Medulloblastoma, Childhood

Eligibility Criteria

Sex

ALL

Age

1 Year - 25 Years

Healthy Volunteers

Not accepted

Interventions

• Liothyronine (L-T3) — DRUG
  Given orally (PO)
• Bevacizumab — DRUG
  Given IV
• Irinotecan — DRUG
  Given IV
• Temozolomide (TMZ) — DRUG
  Given PO

Study Details

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.

Key Dates

Start date

Jul 30, 2026

Status verified

May 2026

Primary completion

Mar 31, 2031

Completion

Mar 31, 2031

Study Design

Enrollment

69 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Phase 1 Cohort (Cohort 1)
  Participants will be treated with a backbone of BIT. L-T3 will be administered on day 1 of each cycle at planned dose levels (DL). Once the Recommended Phase 2 Dose (RP2D) is established with dosing days 1-7, an additional cohort of 6 participants will be treated at the maximum tolerated DL for 14 days of the cycle, Dose level escalation (DLE), which, if tolerated, will become the RP2D. If DLE is not tolerated, the RP2D will become the highest tolerated DL from the prior cohort.
• Experimental: Phase 2 Cohort 1- Relapsed/Progressive Disease
  Participants will be treated at the RP2D of L-T3 based on the results of the safety Phase 1 cohort. 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 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.
• Experimental: Phase 2 Cohort 2 - cfDNA positive in CSF
  Children and young adults with medulloblastoma and CSF cf-DNA positivity without radiographic disease progression/recurrence following standard upfront therapy. will include children and young adults with medulloblastoma and positive cf-DNA in CSF following initial standard therapy and will involve a Phase 2 to evaluate clearance of cf-DNA positive disease in CSF in response to L-T3 monotherapy at the RP2D. Cohort 2 will begin enrolling once the RP2D of L-T3 is established.

Primary Outcome Measure

Proportion of participants experienced an Adverse Event [ Time Frame: Up to 28 days ]

Central Contacts

• Kelly Hitchner
  877-827-3222
  [email protected]

Locations (1)

Find similar trials in San Francisco, CA

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