Trial · NCT04804709
Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)
NCT04804709, Phase 1, Terminated, Columbia University; results first posted 2025-06-08
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG). Trials, record NCT04804709. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04804709
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Non-Invasive Focused Ultrasound (FUS) With Oral Panobinostat in Children With Progressive Diffuse Midline Glioma (DMG)
- NCT number
- NCT04804709
- Status
- Terminated
- Phases
- Phase 1
- Enrollment
- 5 participants, actual
- Lead sponsor
- Columbia University
- Conditions
- Diffuse Intrinsic Pontine Glioma, Diffuse Pontine and Thalamic Gliomas, Diffuse Midline Glioma, H3 K27M-Mutant
- Interventions
- Panobinostat 15 MG, Focused Ultrasound with neuro-navigator-controlled sonication
- First posted
- 2021-03-18
- Last updated
- 2025-06-08
- Results first posted
- 2025-06-08
- Limitations and caveats
- Trial could not be continued due to the manufacturer of Panobinostat discontinuing the drug during the course of the trial.
Source ClinicalTrials.gov
Registered outcomes4
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Adverse Events | Up to 7 months | 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 medication (unrelated, unlikely, possible, probable, and definite). |
| Secondary | Progression Free Survival at 6 Months (PFS6) | Up to 6 months | PFS is defined as the duration of the time from the start of FUS treatment to time of progression or death from any cause, whichever occurs first. Response and progression will be evaluated in this study using the international criteria proposed by the updated Pediatric Response Assessment in Neuro-oncology (RANO) guidelines. |
| Secondary | Overall Survival at 6 Months (OS6) | Up to 7 months | Overall survival is defined as the duration of time from the start of FUS treatment to death from any cause. OS will be measured by follow-up with a study participant every 3-6 months until death for any reason. |
| Secondary | Number of Subjects With Blood Brain Barrier/Tumor Imaging Changes | Up to 8 months | MRI and other radiological evidence to show successful blood brain barrier (BBB) opening and closing will be reviewed and tallied. |
Source ClinicalTrials.gov
Outcomes4
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Adverse Events | Up to 7 months | adverse events | NUMBER | |
| SECONDARY | Progression Free Survival at 6 Months (PFS6) | Up to 6 months | days | MEDIAN | |
| SECONDARY | Overall Survival at 6 Months (OS6) | Up to 7 months | days | MEDIAN | |
| SECONDARY | Number of Subjects With Blood Brain Barrier/Tumor Imaging Changes | Up to 8 months | Participants | COUNT_OF_PARTICIPANTS |
Source ClinicalTrials.gov
Results by group4
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Adverse Events | FUS Using Oral Panobinostat | 31 | adverse events | ||
| Progression Free Survival at 6 Months (PFS6) | FUS Using Oral Panobinostat | 70 | Full Range 16 to 142 | days | |
| Overall Survival at 6 Months (OS6) | FUS Using Oral Panobinostat | 91 | Full Range 70 to 192 | days | |
| Number of Subjects With Blood Brain Barrier/Tumor Imaging Changes | FUS Using Oral Panobinostat | 3 | Participants |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | FUS Using Oral Panobinostat | 3 | |
| Overall Study | COMPLETED | FUS Using Oral Panobinostat | 3 | |
| Overall Study | NOT COMPLETED | FUS Using Oral Panobinostat | 0 |
Source ClinicalTrials.gov
Adverse events31
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 2 | 3 | 11 |
| other | Platelet count decreased | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 3 | 3 | 10 |
| other | Hypokalemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 2 | 3 | 10 |
| other | Lymphocyte count decreased | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 2 | 3 | 20 |
| other | Hypophosphatemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 1 | 3 | 6 |
| other | Hyperglycemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 3 | 3 | 14 |
| other | hypermagnesemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 2 | 3 | 5 |
| other | Hypomagnesemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 1 | 3 | 1 |
| other | Hyperphosphatemia | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 1 | 3 | 1 |
| other | alanine aminotransferase increased | Blood and lymphatic system disorders | FUS Using Oral Panobinostat | 2 | 3 | 8 |
1 to 10 of 31Source ClinicalTrials.gov
See all 31 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | FUS Using Oral Panobinostat | 3 | 3 |
| Total, other adverse events | FUS Using Oral Panobinostat | 3 | 3 |
| Total, serious adverse events | FUS Using Oral Panobinostat | 1 | 3 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04804709
GET https://conductscience.org/api/trials/v1/NCT04804709/events?page=2