Liothyronine + BIT Regimen for Brain Cancer
(PNOC044 Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
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.
Who Is on the Research Team?
Sabine Mueller
Principal Investigator
University of California, San Francisco
Are You a Good Fit for This Trial?
This trial is for children and young adults aged 1-25 with medulloblastoma that has come back or gotten worse after initial treatment. They must have had standard therapy, be in a certain health condition (Karnofsky/Lansky score ≥50), and meet specific blood count and organ function requirements. Some may need to show tumor DNA in their spinal fluid.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive L-T3 in combination with BIT regimen or as monotherapy, with dose escalation to determine RP2D
Follow-up
Participants are monitored for safety and effectiveness after treatment, including cf-DNA clearance and adverse events
Open-label extension (optional)
Participants may continue L-T3 monotherapy for one additional year if benefiting from therapy
What Are the Treatments Tested in This Trial?
Interventions
- BIT Regimen (Bevacizumab, Irinotecan, Temozolomide)
- Liothyronine
Trial Overview
The study tests Liothyronine (L-T3) immunotherapy combined with the BIT chemotherapy regimen (Bevacizumab, Irinotecan, Temozolomide) on those whose medulloblastoma has relapsed or progressed post-standard therapy. It's divided into phases to assess safety and effectiveness.
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
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.
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.
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.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sabine Mueller, MD, PhD
Lead Sponsor
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