NgFUS NIVO: NeuroNavigation-Guided Focused Ultrasound With Nivolumab in Relapsed and Progressive DMG and Other High Grade Brain Tumors

Sponsor
Children's National Research Institute
Study ID
NCT07664176
Phase
PHASE1
Status
Not Yet Recruiting

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Conditions

  • Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
  • Brain Tumor Recurrent
  • Diffuse Midline Glioma or Diffuse Intrinsic Pontine Glioma
  • Ependymoma Recurrent
  • High Grade Gliomas
  • Medulloblastoma Recurrent

Eligibility Criteria

Sex
ALL
Age
3 Years - 25 Years
Healthy Volunteers
Not accepted

Interventions

  • NeuroNavigation-Guided Focused Ultrasound (NgFUS) — DEVICE
    NeuroNavigation-Guided Focused Ultrasound, every 2 or 4 weeks for 3 cycles of 28 days
  • Lumason — DRUG
    Lumason is a sulfur hexafluoride microsphere ultrasound contrast agent which will be used as a mechanical resonator. Lumason will be given in combination with NgFUS every 2 or 4 weeks for 3 cycles of 28 days.
  • Nivolumab — DRUG
    Nivolumab is a monoclonal antibody targeting the immune checkpoint axis PD-1/PD-L1. Nivolumab will be given prior to NgFUS with microbubbles (Lumason) every 2 or 4 weeks for 3 cycles of 28 days.

Study Details

This is an open-label phase 1 safety and feasibility study evaluating a novel combination therapy for progressive and relapsed diffuse midline glioma (DMG) and other progressive and relapsed high-grade brain tumors. This study combines intravenous nivolumab therapy infused following transient blood-brain barrier opening (BBBO) using low-intensity focused ultrasound with microbubble (LIFU-MB) treatment using NeuroNavigation-Guided Focused Ultrasound (NgFUS). There are two groups in this study: * Group A: Patients with relapsed or progressive diffuse midline glioma in the brainstem * Group B: Patients with relapsed or progressive high grade brain tumor that clinically require surgical resection The primary outcome is to evaluate the safety and feasibility of 3 cycles of nivolumab with BBB disruption using NgFUS with microbubbles in pediatric patients with progressive or relapsed brainstem DMG or with high grade brain tumors after surgery. Secondary outcomes include preliminary efficacy and immunological effects.

Key Dates

First listed
Jun 24, 2026
Start date
Jun 1, 2026
Status verified
Jun 2026
Primary completion
Dec 31, 2030
Completion
Dec 31, 2030

Study Design

Enrollment
30 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: Group A: Patients with relapsed or progressive diffuse midline glioma in the brainstem
    Patients with relapsed or progressive diffuse midline glioma in the brainstem, receiving nivolumab with NgFUS either every 2 weeks or every 4 weeks.
  • Experimental: Group B: Patients with relapsed or progressive high grade brain tumors requiring surgical resection
    Patients with relapsed or progressive high grade brain tumors that clinically require surgical resection, receiving one dose of NgFUS prior to surgery, and nivolumab with NgFUS every 4 weeks following recovery from surgery.

Primary Outcome Measure

Incidence of dose limiting toxicities (DLT) as assessed by CTCAE v6.0. [ Time Frame: First treatment through 28 days post-first treatment. ]

Central Contacts

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