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Sound waves may help chemo reach deadly childhood brain tumors

NCT ID NCT05630209

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests whether a special ultrasound device can safely open the blood-brain barrier in children with a rare, aggressive brain tumor called DIPG. By temporarily allowing the chemotherapy drug doxorubicin to reach the tumor, researchers hope to improve treatment. The study involves 10 children aged 5 to 21 and focuses on safety and how well the barrier opens.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 10 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2023

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

5 to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age between 5 and 21 years, inclusive. Subjects younger than 5 years old may be eligible after discussion with the Sponsor Medical Monitor/designee (i.e. DSMB). * Patient diagnosed with DIPG * At least 4-week and not greater than 12 weeks from completion of radiation therapy * Post-radiation imaging does not show evidence of necrosis/ hemorrhage or other features that contraindicate MRgFUS * If brain surgery occurred, at least 14 days passed since last brain surgery and the patient is fully recovered and neurologically stable * If on steroids, stable or decreasing dose for at least 7 days prior to study entry * Stable or improving neurologic status for 7 days prior to study entry * Able and willing to give consent and/or assent or have a legal guardian who is able and willing to do so * Able to attend all study visits and with life expectancy of at least 6 months Exclusion Criteria: * Tumor not visible on any pre-therapy or post-radiation imaging * Previous participation in other chemotherapy, molecularly targeted therapy, or immunotherapy treatment-related phase 1 or 2 trials * Symptoms and signs of increased intracranial pressure * Subject with metastatic disease * Subject with ventricular peritoneal shunt * Subject receiving bevacizumab (Avastin) therapy or increasing doses of steroids * Anti-coagulant therapy, or medications known to increase risk of hemorrhage, (e.g., ASA, non-steroidal anti-inflammatory drugs \[NSAIDs\], statins) within washout period prior to treatment * History of a bleeding disorder, coagulopathy or with a history of spontaneous tumor hemorrhage. * Hypertension per age * Cerebral or systemic vasculopathy, including intracranial thrombosis, vascular malformation, cerebral aneurysm, or vasculitis * Immunosuppression (corticosteroids to prevent/treat brain edema are permitted). * Patients with positive HIV status * Active seizure disorder or epilepsy (seizures despite medical treatment) for a minimum of 4 weeks prior to first cycle/Exablate BBBD procedure captured by history * Known sensitivity to gadolinium-based contrast agents * Known sensitivity to DEFINITY® ultrasound contrast agent or known hypersensitivity to perflutren microsphere or its components, e.g., polyethylene glycol * Contraindication to Doxorubicin * Previous treatment with complete cumulative doses of Doxorubicin, daunorubicin, idarubicin, and/or other anthracyclines and anthracenediones * Severely impaired renal function with estimated glomerular filtration rate \<2 standard deviations for age * Patients that may require trastuzumab during the study * Patients that may require inhibitors and inducers of CYP3A4, CYP2D6, and/or P-gp during the study

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Cook Children's Health Care System

    Fort Worth, Texas, 76104, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

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