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Sound waves open brain barrier to attack deadly childhood tumors

NCT ID NCT05762419

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early study tests whether focused ultrasound can safely open the blood-brain barrier in children with progressive diffuse midline glioma (DMG), a rare and aggressive brain tumor. Ten children aged 4–21 will receive oral etoposide chemotherapy after ultrasound treatment to allow more drug to reach the tumor. The goal is to see if the procedure is safe and feasible, not yet to measure effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Etoposide (oral chemotherapy) combined with focused ultrasound to open the blood-brain barrier
What this could lead to
If successful, this approach could improve drug delivery to brain tumors, potentially slowing disease progression in children with diffuse midline glioma.
What could go wrong
This is a very early, small feasibility study (10 children). It primarily tests safety and whether the blood-brain barrier can be opened—not yet whether it improves outcomes. Risks include side effects from etoposide and the ultrasound procedure.

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

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 10 people

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

Started

Jun 2023

Expected to finish

Dec 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

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

Ages

4 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: * Ages 4 - 21 years * Radiological diagnosis of Diffuse Midline Glioma with tumor involving the pons (intrinsic, pontine based infiltrative lesion; hypointense on T1 weighted images (T1WIs) and hyperintense in T2 sequences, with mass effect on the adjacent structures and occupying at least 50% of the pons), thalami, and/or histological confirmation of H3K27M mutation of pontine or thalamic glioma. Subjects must have evidence of clinical and/or radiographic progression of disease. * Lansky performance status score of at least 60 for subjects 16 years of age or younger. * Karnofsky performance status of at least 60 for subjects greater than 16 years of age * Organ Function: * Adequate hematologic function defined as: * Peripheral absolute neutrophil count ≥ 1,500/µL * Platelet count ≥ 100,000/µL * Partial thromboplastin time (PTT) and activated partial thromboplastin time (APTT): within normal institutional limits * Adequate renal function defined as: * Potassium and magnesium levels within institutional limits * Serum creatinine below the institutional upper limit of normal (ULN) for age and gender, or creatinine clearance: ≥ 60 mL/min/1.73m2 * Adequate hepatic function defined as: * Total bilirubin below the institutional ULN for age * Aspartate aminotransferase (AST)/alanine aminotransferase (ALT) ≤ 2.5 × institutional ULN * Prior Therapy: * Subjects must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment. * Cytotoxic chemotherapy or anti-cancer agents known to be myelosuppressive: at least 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy. * Anti-cancer agents not known to be myelosuppressive: at least 7 days must have elapsed from last dose of agent. * Antibodies: at least 21 days must have elapsed from infusion of last dose of antibody. * Interleukins, interferons, and cytokines: at least 21 days must have elapsed since the completion of interleukins, interferon, or cytokines. * Stem cell infusions: at least 42 days must have elapsed after completion of an autologous stem cell infusion, and at least 84 days must have elapsed after completion of an allogeneic stem cell infusion. * Cellular therapy: at least 42 days must have elapsed since the completion of any type of cellular therapy * Radiotherapy (XRT): at least 1 month must have elapsed after local XRT. * Subjects must be on a stable or decreasing dose of steroids, as well as stable dose of anti-seizure medication for at least 1 week. * Subject able to give consent Exclusion Criteria: * Subjects that have previously received etoposide therapy * Subjects unable to tolerate study procedures and/or anesthesia based on the opinion of the principal investigator * Uncontrolled seizure disorder * Pregnancy or Breast-Feeding: pregnant or breast-feeding women will not be entered on this study, since there is yet no available information regarding human fetal or teratogenic toxicities; a pregnancy test must be obtained in girls who are post-menarchal. Males with female partners of reproductive potential or females of reproductive potential may not participate unless they have agreed to use two effective methods of birth control- including a medically accepted barrier method of contraception (e.g., a male or female condom) for the entire period in which they are receiving protocol therapy and for at least 1 month following their last study treatment requirement. Abstinence is an acceptable method of birth control. Women of childbearing potential will be provided a routine quantitative beta-human chorionic gonadotropin (B-hCG) test during the pre-study phase, prior to enrollment and each cycle. * Concomitant medications: subjects who are currently receiving another investigational drug or other anti-cancer agents are not eligible. * Screening EKG with a QTc \> 450 msec. * Subjects with evidence of active systemic infection * Subjects with a documented allergy to compounds of similar chemical or biologic composition to etoposide or gadolinium compounds * Subjects with implanted metallic or electrical devices * Subjects with uncontrollable hypertension * Subjects with a documented bleeding disorder * Subjects with history of structural cardiac anomalies or arrhythmias * Subjects with history of unprovoked stroke or signs of stroke in the area of FUS target * Subjects with SARS-CoV-2 infection requiring hospitalization in the past month and requires anticoagulation as per the Columbia University Irving Medical Center (CUIMC) institutional "Anticoagulation for COVID-19 Positive Pediatric Inpatients" guidelines (See Appendix B) * Subjects with coagulopathy or under anticoagulant therapy. * Subjects with signs of impending herniation or an acute or previous intratumoral hemorrhage * Subjects with spinal cord diffuse midline glioma * Subjects receiving a drug where CNS toxicity is reasonably suspected

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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.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Locations

  • Columbia University Irving Medical Center

    RECRUITING

    New York, New York, 10032, United States

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