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NCT04804709 · ClinicalTrials.gov registry record · Phase 1

Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)

A Phase 1 study, sponsored by Columbia University.

Terminated
Registry status
Phase 1
Development phase
5
Enrollment target

NCT04804709: Clinical Trial Phase 1 study, sponsored by Columbia University.

NCT04804709 is a Phase 1 study that was terminated before completion, run by Columbia University. The registered enrollment target is 5 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04804709, a Phase 1 study, was terminated before completion, sponsored by Columbia University.

TERMINATED
Registry status
Phase 1
Development phase
5 participants
Enrollment target

Study Summary

The blood brain barrier (BBB) prevents some drugs from successfully reaching the target tumor. Focused Ultrasound (FUS) using microbubbles and neuro-navigator-controlled sonication is a non-invasive method of temporarily opening up the blood brain barrier to allow a greater concentration of the drug to reach into the brain tumor. This may improve response and may also reduce system side effects in the patient. The primary purpose of this study is to evaluate the feasibility of safely opening the BBB in children with progressive diffuse midline gliomas (DMG) treated with oral Panobinostat using FUS with microbubbles and neuro-navigator-controlled sonication. For the purpose of the study, the investigators will be opening up the BBB temporarily in one, two, or three locations around the tumor using the non-invasive FUS technology, and administrating oral Panobinostat in children with progressive DMG.

Primary Outcome

Safety will be assessed by evaluation of physical and neurologic examinations, laboratory studies, radiographic studies, and by adverse events as per the CTCAE version 5.0. An adverse event is any new or worsening symptom or clinical finding which occurs during the study period. Adverse events are to be recorded irrespective of causality on the adverse event form. Each event will be described by its severity (mild, moderate, severe, life-threatening), duration, and relation to the study medicati

Interventions

  • DRUG Panobinostat 15 MG
  • DEVICE Focused Ultrasound with neuro-navigator-controlled sonication

Trial Details

FieldValue
Enrollment Target 5 participants
Start Date 2021-07-28
Est. Completion 2022-03-31
Phase Phase 1
Columbia University

958 total trials

Why NCT04804709 stopped before completion

NCT04804709 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (92% lower).

The record links to 0 conditions, and to 2 interventions - of which Panobinostat 15 MG is the first listed.

NCT04804709 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT04804709 about?

NCT04804709 is a clinical study titled "Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)". The blood brain barrier (BBB) prevents some drugs from successfully reaching the target tumor. Focused Ultrasound (FUS) using microbubbles and neuro-navigator-controlled sonication is a non-invasive method of temporarily opening up the blood brain barrier to allow a greater concentration of the drug...

What is the current status of trial NCT04804709?

This trial is currently terminated. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2021-07-28. Estimated completion is 2022-03-31.

What interventions are being tested in trial NCT04804709?

The interventions under investigation include: Panobinostat 15 MG (DRUG), Focused Ultrasound with neuro-navigator-controlled sonication (DEVICE).

Who is sponsoring clinical trial NCT04804709?

This trial is sponsored by Columbia University, which has 958 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT04804709's enrollment target sits among peer trials

5 1989th of 2000 higher than 6 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT04804709, the US trial registry maintained by the National Library of Medicine. NCT04804709 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.