Trial · NCT02717455
Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma
NCT02717455, Phase 1, Completed, Pediatric Brain Tumor Consortium; results first posted 2024-04-25
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma. Trials, record NCT02717455. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02717455
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma
- NCT number
- NCT02717455
- Status
- Completed
- Phases
- Phase 1
- Enrollment
- 53 participants, actual
- Lead sponsor
- Pediatric Brain Tumor Consortium
- Conditions
- Glioma
- Interventions
- LBH589
- First posted
- 2016-03-23
- Last updated
- 2024-04-25
- Results first posted
- 2024-04-25
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | 4 weeks | DLTs were defined as adverse events that were at least possibly related to panobinostat that occurred during the first 4 weeks of therapy regardless of expectedness. Hematologic DLTs included grade 4 thrombocytopenia, grade 3 thrombocytopenia with bleeding, grade 3 thrombocytopenia that occurs twice within a treatment course, myelosuppression that causes greater than a 14-day delay between treatment courses, grade 4 neutropenia, grade 3 or 4 febrile neutropenia. Non-hematologic DLTs included any grade 3 or greater non-hematologic toxicities with a few exclusions (such as grade 3 nausea/vomiting that is responsive to antiemetics and that resolves to grade 2 or lower within 5 days, etc.), any grade 2 non-hematological toxicity that persists for more than 7 days and is considered sufficiently medically significant or sufficiently intolerable by patients, and any panobinostat-related non-hematological toxicity that results in a delay of treatment \> 14 days between treatment courses. |
| Primary | Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 1 | 4 weeks | The MTD of panobinostat was defined as the dose at which the continual reassessment method (CRM) estimated that 25% of patients were expected to experience DLTs. Stratum 1 consisted of recurrent or progressive diffuse intrinsic pontine glioma (DIPG) patients who were treated with the "3 times/week, three weeks on, one week off" schedule (1 course = 28 days). |
| Primary | Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 2 | 4 weeks | The MTD of panobinostat was defined as the dose at which the continual reassessment method (CRM) estimated that 25% of patients were expected to experience DLTs. For Stratum 2, non-progressed DIPG or H3K27M+ thalamic diffuse malignant glioma (DMG) patients who completed conventional radiation treatment were eligible. All patients enrolled on this stratum had DIPG tumors and were treated with the "3 times/week, every other week" schedule (1 course = 28 days). |
| Primary | Volume of Distribution (Vd) | Up to day 3 | Plasma samples for pharmacokinetic (PK) analysis were drawn at pre-dose and at 0.5, 1, 2, 4, 8 (±1), 24 (±4) hours after the first dose of panobinostat, as well as prior to the second dose on Course 1 Day 3. Volume of distribution (Vd) was estimated using a noncompartmental method. |
| Primary | Elimination Rate (Kel) | Up to day 3 | Plasma samples for pharmacokinetic (PK) analysis were drawn at pre-dose and at 0.5, 1, 2, 4, 8 (±1), 24 (±4) hours after the first dose of panobinostat, as well as prior to the second dose on Course 1 Day 3. Elimination rate (Kel) was estimated using a noncompartmental method. |
| Primary | Half-life (t1/2) | Up to day 3 | Plasma samples for pharmacokinetic (PK) analysis were drawn at pre-dose and at 0.5, 1, 2, 4, 8 (±1), 24 (±4) hours after the first dose of panobinostat, as well as prior to the second dose on Course 1 Day 3. Half-life (t1/2) was estimated using a noncompartmental method. |
| Primary | Clearance (CL/F) | Up to day 3 | Plasma samples for pharmacokinetic (PK) analysis were drawn at pre-dose and at 0.5, 1, 2, 4, 8 (±1), 24 (±4) hours after the first dose of panobinostat, as well as prior to the second dose on Course 1 Day 3. Clearance (CL/F) was estimated using a noncompartmental method. |
| Primary | Area Under the Curve (AUC) | Up to day 3 | Plasma samples for pharmacokinetic (PK) analysis were drawn at pre-dose and at 0.5, 1, 2, 4, 8 (±1), 24 (±4) hours after the first dose of panobinostat, as well as prior to the second dose on Course 1 Day 3. The area under the curve (AUC) was estimated using a noncompartmental method and calculated from time of dosing to the last measurable concentration. |
| Secondary | Progression-free Survival (PFS) in Stratum 1 | From date on treatment until date of PD or death due to any cause or date of last follow-up | PFS was measured from the time of treatment initiation until the time of progressive disease (PD) or death due to any cause for patients with an event, or until the time of last follow-up for patients who were progression free. |
| Secondary | Overall Survival (OS) in Stratum 1 | From date on treatment until date of death due to any cause or date of last follow-up | OS was measured from the time of treatment initiation until the time of death due to any cause for patients who died, or until the time of last follow-up for patients who survived. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | 4 weeks | Patients who were evaluable for DLT assessment were included in this analysis. Of all 53 patients enrolled, 7 patients were not evaluable for DLT assessment: 4 patients received less than required dose of study drug, 2 patients did not start protocol therapy, and 1 patient had insufficient labs to monitor for DLTs. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 1 | 4 weeks | Patients who were enrolled on stratum 1 and were evaluable for dose finding assessment were used to determine the MTD for stratum 1. Of 19 patients enrolled on stratum 1, 2 patients were not evaluable for dose finding assessment due to receiving less than required dose of study drug. The remaining 17 patients were used to determine the MTD for stratum I. | mg/m^2/day | NUMBER |
| PRIMARY | Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 2 | 4 weeks | Patients who were enrolled on stratum 2 and were evaluable for dose finding assessment were used to determine the MTD for stratum 2. Of 34 patients enrolled on stratum 2, 5 patients were not evaluable due to: receiving less than required dose of study drug (2), disease progression before active treatment (1), withdrawal/refusal prior to protocol therapy (1), and insufficient labs to monitor for dose-limiting toxicities (1). The remaining 29 patients were used to determine the MTD for stratum 2. | mg/m^2/day | NUMBER |
| PRIMARY | Volume of Distribution (Vd) | Up to day 3 | Patients who had PK samples collected and had Vd data available were included in this analysis. | L | MEAN |
| PRIMARY | Elimination Rate (Kel) | Up to day 3 | Patients who had PK samples collected and had Kel data available were included in this analysis. | per hour | MEAN |
| PRIMARY | Half-life (t1/2) | Up to day 3 | Patients who had PK samples collected and had t1/2 data available were included in this analysis. | hour | MEAN |
| PRIMARY | Clearance (CL/F) | Up to day 3 | Patients who had PK samples collected and had CL/F data available were included in this analysis. | L/h | MEAN |
| PRIMARY | Area Under the Curve (AUC) | Up to day 3 | Patients who had PK samples collected and had AUC data available were included. | h*ng/mL | MEAN |
| SECONDARY | Progression-free Survival (PFS) in Stratum 1 | From date on treatment until date of PD or death due to any cause or date of last follow-up | Recurrent/progressive DIPG patients who were enrolled on stratum 1 and received at least one dose of panobinostat were included in the analysis. Per the associated protocol objective, all dose levels on stratum 1 were combined to estimate the PFS for this stratum. | months | MEDIAN |
| SECONDARY | Overall Survival (OS) in Stratum 1 | From date on treatment until date of death due to any cause or date of last follow-up | Recurrent/progressive DIPG patients who were enrolled on stratum 1 and received at least one dose of panobinostat were included in the analysis. Per the associated protocol objective, all dose levels on stratum 1 were combined to estimate the OS for this stratum. | months | MEDIAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group56
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 1, Dose Level 1 (10 mg/m^2) | 0 | Participants | ||
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 1, Dose Level 2 (16 mg/m^2) | 2 | Participants | ||
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 2, Dose Level 1 (16 mg/m^2) | 0 | Participants | ||
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 2, Dose Level 2 (22 mg/m^2) | 1 | Participants | ||
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 2, Dose Level 3 (28 mg/m^2) | 4 | Participants | ||
| Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) | Stratum 2, Dose Level 4 (36 mg/m^2) | 3 | Participants | ||
| Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 1 | Stratum 1 | 10 | mg/m^2/day | ||
| Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 2 | Stratum 2 | 22 | mg/m^2/day | ||
| Volume of Distribution (Vd) | Stratum 1, Dose Level 1 (10 mg/m^2) | 1704 | Standard Deviation 827 | L | |
| Volume of Distribution (Vd) | Stratum 1, Dose Level 2 (16 mg/m^2) | 2135 | Standard Deviation 1268 | L |
1 to 10 of 56Source ClinicalTrials.gov
See all 56 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Stratum 1, Dose Level 1 (10 mg/m^2) | 13 | |
| Overall Study | STARTED | Stratum 1, Dose Level 2 (16 mg/m^2) | 6 | |
| Overall Study | STARTED | Stratum 2, Dose Level 1 (16 mg/m^2) | 3 | |
| Overall Study | STARTED | Stratum 2, Dose Level 2 (22 mg/m^2) | 12 | |
| Overall Study | STARTED | Stratum 2, Dose Level 3 (28 mg/m^2) | 12 | |
| Overall Study | STARTED | Stratum 2, Dose Level 4 (36 mg/m^2) | 7 | |
| Overall Study | COMPLETED | Stratum 1, Dose Level 1 (10 mg/m^2) | 10 | |
| Overall Study | COMPLETED | Stratum 1, Dose Level 2 (16 mg/m^2) | 5 | |
| Overall Study | COMPLETED | Stratum 2, Dose Level 1 (16 mg/m^2) | 3 | |
| Overall Study | COMPLETED | Stratum 2, Dose Level 2 (22 mg/m^2) | 6 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events666
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 | Stratum 1, Dose Level 1 (10 mg/m^2) | 3 | 13 | 3 |
| other | Anemia | Blood and lymphatic system disorders | Stratum 1, Dose Level 2 (16 mg/m^2) | 0 | 6 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Stratum 2, Dose Level 1 (16 mg/m^2) | 0 | 3 | 0 |
| other | Anemia | Blood and lymphatic system disorders | Stratum 2, Dose Level 2 (22 mg/m^2) | 3 | 11 | 6 |
| other | Anemia | Blood and lymphatic system disorders | Stratum 2, Dose Level 3 (28 mg/m^2) | 7 | 12 | 16 |
| other | Anemia | Blood and lymphatic system disorders | Stratum 2, Dose Level 4 (36 mg/m^2) | 3 | 6 | 7 |
| other | Cardiac disorders - other, specify | Cardiac disorders | Stratum 1, Dose Level 1 (10 mg/m^2) | 0 | 13 | 0 |
| other | Cardiac disorders - other, specify | Cardiac disorders | Stratum 1, Dose Level 2 (16 mg/m^2) | 0 | 6 | 0 |
| other | Cardiac disorders - other, specify | Cardiac disorders | Stratum 2, Dose Level 1 (16 mg/m^2) | 0 | 3 | 0 |
| other | Cardiac disorders - other, specify | Cardiac disorders | Stratum 2, Dose Level 2 (22 mg/m^2) | 0 | 11 | 0 |
1 to 10 of 666Source ClinicalTrials.gov
See all 666 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Stratum 1, Dose Level 1 (10 mg/m^2) | 13 | 13 |
| Total, all-cause mortality | Stratum 1, Dose Level 2 (16 mg/m^2) | 5 | 6 |
| Total, all-cause mortality | Stratum 2, Dose Level 1 (16 mg/m^2) | 3 | 3 |
| Total, all-cause mortality | Stratum 2, Dose Level 2 (22 mg/m^2) | 5 | 11 |
| Total, all-cause mortality | Stratum 2, Dose Level 3 (28 mg/m^2) | 11 | 12 |
| Total, all-cause mortality | Stratum 2, Dose Level 4 (36 mg/m^2) | 5 | 6 |
| Total, other adverse events | Stratum 1, Dose Level 1 (10 mg/m^2) | 13 | 13 |
| Total, other adverse events | Stratum 1, Dose Level 2 (16 mg/m^2) | 6 | 6 |
| Total, other adverse events | Stratum 2, Dose Level 1 (16 mg/m^2) | 3 | 3 |
| Total, other adverse events | Stratum 2, Dose Level 2 (22 mg/m^2) | 11 | 11 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| UM1CA081457 | Pediatric Brain Tumor Consortium A multi-institutional consortium devoted to novel phase I and II clinical evaluations of experimental treatment approaches for pediatric CNS tumors | ONAR-THOMAS, ARZU; DUNKEL, IRA J | ST. JUDE CHILDREN'S RESEARCH HOSPITAL | NATIONAL CANCER INSTITUTE | 2014 to 2025 |
Source NIH RePORTER
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.
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GET https://conductscience.org/api/trials/v1/NCT02717455/results?page=2