NCT02717455 (PBTC-047) is a Phase 1 clinical trial of LBH589 in Glioma, sponsored by Pediatric Brain Tumor Consortium.

Who is sponsoring NCT02717455?

NCT02717455 is sponsored by Pediatric Brain Tumor Consortium.

What drugs are being tested in NCT02717455?

Interventions in NCT02717455: LBH589.

Is NCT02717455 still recruiting?

NCT02717455 is currently completed. Last updated 25 April 2024.

Are results published for NCT02717455?

Yes — primary outcome results have been posted to ClinicalTrials.gov. See the outcomes section above for details.

Last reviewed 2024-04-25 · How we verify

NCT02717455: PBTC-047

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Trial of Panobinostat in Children With Diffuse Intrinsic Pontine Glioma

Completed Phase 1 Results posted Last updated 25 April 2024

What this trial tests

Phase 1 trial testing LBH589 in Glioma in 53 participants. Completed in 31 March 2024.

Timeline

28 June 2016

Primary endpoint
14 February 2022

31 March 2024

Quick facts

Lead sponsor	Pediatric Brain Tumor Consortium
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	53
Start date	28 June 2016
Primary completion	14 February 2022
Estimated completion	31 March 2024
Sites	10 locations across United States

Drugs / interventions tested

LBH589 — full drug profile →

Conditions studied

Glioma — all drugs for Glioma →

Sponsor

Pediatric Brain Tumor Consortium

Who can join

Adults 2 to 21, any sex, with Glioma. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Patients Who Experienced Dose Limiting Toxicities (DLTs) Primary · 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/vomitin

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	0
Stratum 1, Dose Level 2 (16 mg/m^2)	2
Stratum 2, Dose Level 1 (16 mg/m^2)	0
Stratum 2, Dose Level 2 (22 mg/m^2)	1
Stratum 2, Dose Level 3 (28 mg/m^2)	4
Stratum 2, Dose Level 4 (36 mg/m^2)	3

Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 1 Primary · 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).

Group	Value	95% CI
Stratum 1	10

Maximum Tolerated Dose (MTD) of Panobinostat in Stratum 2 Primary · 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).

Group	Value	95% CI
Stratum 2	22

Volume of Distribution (Vd) Primary · 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.

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	1704	± 827
Stratum 1, Dose Level 2 (16 mg/m^2)	2135	± 1268
Stratum 2, Dose Level 1 (16 mg/m^2)	3358	± 1136
Stratum 2, Dose Level 2 (22 mg/m^2)	2141	± 906
Stratum 2, Dose Level 3 (28 mg/m^2)	1967	± 455
Stratum 2, Dose Level 4 (36 mg/m^2)	2487	± 2760

Elimination Rate (Kel) Primary · 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.

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	0.059	± 0.024
Stratum 1, Dose Level 2 (16 mg/m^2)	0.065	± 0.013
Stratum 2, Dose Level 1 (16 mg/m^2)	0.047	± 0.003
Stratum 2, Dose Level 2 (22 mg/m^2)	0.057	± 0.012
Stratum 2, Dose Level 3 (28 mg/m^2)	0.047	± 0.006
Stratum 2, Dose Level 4 (36 mg/m^2)	0.066	± 0.017

Half-life (t1/2) Primary · 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.

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	13.0	± 4.0
Stratum 1, Dose Level 2 (16 mg/m^2)	11.0	± 2.0
Stratum 2, Dose Level 1 (16 mg/m^2)	14.7	± 1.1
Stratum 2, Dose Level 2 (22 mg/m^2)	12.6	± 2.8
Stratum 2, Dose Level 3 (28 mg/m^2)	14.8	± 1.9
Stratum 2, Dose Level 4 (36 mg/m^2)	11.3	± 3.8

Clearance (CL/F) Primary · 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.

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	87	± 19
Stratum 1, Dose Level 2 (16 mg/m^2)	146	± 104
Stratum 2, Dose Level 1 (16 mg/m^2)	156	± 44
Stratum 2, Dose Level 2 (22 mg/m^2)	115	± 34
Stratum 2, Dose Level 3 (28 mg/m^2)	93	± 26
Stratum 2, Dose Level 4 (36 mg/m^2)	129	± 109

Area Under the Curve (AUC) Primary · 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.

Group	Value	95% CI
Stratum 1, Dose Level 1 (10 mg/m^2)	102	± 44
Stratum 1, Dose Level 2 (16 mg/m^2)	191	± 146
Stratum 2, Dose Level 1 (16 mg/m^2)	143	± 64
Stratum 2, Dose Level 2 (22 mg/m^2)	239	± 71
Stratum 2, Dose Level 3 (28 mg/m^2)	284	± 73
Stratum 2, Dose Level 4 (36 mg/m^2)	372	± 222

Progression-free Survival (PFS) in Stratum 1 Secondary · 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.

Group	Value	95% CI
Stratum 1	1.87	0.73 – 9.00

Overall Survival (OS) in Stratum 1 Secondary · 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.

Group	Value	95% CI
Stratum 1	5.23	0.73 – 36.60

Progression-free Survival (PFS) in Stratum 2 Secondary · From date on treatment until date of PD or death due to any cause or date of last follow-up

Group	Value	95% CI
Stratum 2	3.83	0.23 – 14.93

Overall Survival (OS) in Stratum 2 Secondary · 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.

Group	Value	95% CI
Stratum 2	9.13	0.23 – 20.30

Adverse events — posted to ClinicalTrials.gov

Time frame: Approximately 2 years after initiation of protocol treatment. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Stratum 1, Dose Level 1 (10 mg/m^2)

Serious: 9/13 (69%)

Deaths: 13/13

Stratum 1, Dose Level 2 (16 mg/m^2)

Serious: 4/6 (67%)

Deaths: 5/6

Stratum 2, Dose Level 1 (16 mg/m^2)

Serious: 0/3 (0%)

Deaths: 3/3

Stratum 2, Dose Level 2 (22 mg/m^2)

Serious: 5/11 (45%)

Deaths: 5/11

Stratum 2, Dose Level 3 (28 mg/m^2)

Serious: 4/12 (33%)

Deaths: 11/12

Stratum 2, Dose Level 4 (36 mg/m^2)

Serious: 4/6 (67%)

Deaths: 5/6

Serious adverse events (32 terms)

Reaction	System	Stratum 1, Dose Level 1 (1…	Stratum 1, Dose Level 2 (1…	Stratum 2, Dose Level 1 (1…	Stratum 2, Dose Level 2 (2…	Stratum 2, Dose Level 3 (2…	Stratum 2, Dose Level 4 (3…
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - other, specify	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—
Respiratory failure	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—	—	—
Platelet count decreased	Investigations	—	—	—	—	—	—
Depressed level of consciousness	Nervous system disorders	—	—	—	—	—	—
Hydrocephalus	Nervous system disorders	—	—	—	—	—	—
Nervous system disorders - other, specify	Nervous system disorders	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Dysphagia	Gastrointestinal disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—
Gait disturbance	General disorders	—	—	—	—	—	—
Catheter related infection	Infections and infestations	—	—	—	—	—	—
Sepsis	Infections and infestations	—	—	—	—	—	—
Urinary tract infection	Infections and infestations	—	—	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Generalized muscle weakness	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Muscle weakness left-sided	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Muscle weakness right-sided	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Trismus	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Ataxia	Nervous system disorders	—	—	—	—	—	—
Dizziness	Nervous system disorders	—	—	—	—	—	—
Dysarthria	Nervous system disorders	—	—	—	—	—	—

Other adverse events (79 terms — click to expand)

Reaction	System	Stratum 1, Dose Level 1 (1…	Stratum 1, Dose Level 2 (1…	Stratum 2, Dose Level 1 (1…	Stratum 2, Dose Level 2 (2…	Stratum 2, Dose Level 3 (2…	Stratum 2, Dose Level 4 (3…
Platelet count decreased	Investigations	—	—	—	—	—	—
White blood cell decreased	Investigations	—	—	—	—	—	—
Lymphocyte count decreased	Investigations	—	—	—	—	—	—
Diarrhea	Gastrointestinal disorders	—	—	—	—	—	—
Vomiting	Gastrointestinal disorders	—	—	—	—	—	—
Alanine aminotransferase increased	Investigations	—	—	—	—	—	—
Anemia	Blood and lymphatic system disorders	—	—	—	—	—	—
Nausea	Gastrointestinal disorders	—	—	—	—	—	—
Neutrophil count decreased	Investigations	—	—	—	—	—	—
Hypoalbuminemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypophosphatemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Fatigue	General disorders	—	—	—	—	—	—
Aspartate aminotransferase increased	Investigations	—	—	—	—	—	—
Hypertension	Vascular disorders	—	—	—	—	—	—
Constipation	Gastrointestinal disorders	—	—	—	—	—	—
Hemoglobin increased	Investigations	—	—	—	—	—	—
Investigations - other, specify	Investigations	—	—	—	—	—	—
Weight loss	Investigations	—	—	—	—	—	—
Anorexia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypermagnesemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypoglycemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypokalemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Metabolism and nutrition disorders - other, specify	Metabolism and nutrition disorders	—	—	—	—	—	—
Abdominal pain	Gastrointestinal disorders	—	—	—	—	—	—
Gastrointestinal disorders - other, specify	Gastrointestinal disorders	—	—	—	—	—	—
Alkaline phosphatase increased	Investigations	—	—	—	—	—	—
Blood bilirubin increased	Investigations	—	—	—	—	—	—
Creatinine increased	Investigations	—	—	—	—	—	—
Electrocardiogram qt corrected interval prolonged	Investigations	—	—	—	—	—	—
Dehydration	Metabolism and nutrition disorders	—	—	—	—	—	—
Hyperglycemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Hypocalcemia	Metabolism and nutrition disorders	—	—	—	—	—	—
Generalized muscle weakness	Musculoskeletal and connective tissue disorders	—	—	—	—	—	—
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - other, specify	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—	—	—	—	—	—
Ataxia	Nervous system disorders	—	—	—	—	—	—
Headache	Nervous system disorders	—	—	—	—	—	—
Insomnia	Psychiatric disorders	—	—	—	—	—	—
Dyspnea	Respiratory, thoracic and mediastinal disorders	—	—	—	—	—	—
Skin and subcutaneous tissue disorders - other, specify	Skin and subcutaneous tissue disorders	—	—	—	—	—	—
Cardiac disorders - other, specify	Cardiac disorders	—	—	—	—	—	—

Most-reported serious reactions: Neoplasms benign, malignant and unspecified (incl cysts and polyps) - other, specify, Respiratory failure, Neutrophil count decreased, Platelet count decreased, Depressed level of consciousness, Hydrocephalus, Nervous system disorders - other, specify, Hypertension.

Data from ClinicalTrials.gov NCT02717455 adverse events section.

Sponsor's own description

This phase I trial studies the side effects and best dose of panobinostat in treating younger patients with diffuse intrinsic pontine glioma (DIPG). Panobinostat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Stratum 1 treats patients with DIPG that has returned or gotten worse (progressed). Stratum 2 treats patients with DIPG or H3K27+Thalamic Diffuse Malignant Glioma (DMG) that has not yet gotten worse.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Transcriptional Dependencies in Diffuse Intrinsic Pontine Glioma.
Nagaraja S, Vitanza NA, Woo PJ, Taylor KR, et al · · 2017 · cited 323× · PMID 28434841 · DOI 10.1016/j.ccell.2017.03.011
Pervasive H3K27 Acetylation Leads to ERV Expression and a Therapeutic Vulnerability in H3K27M Gliomas.
Krug B, De Jay N, Harutyunyan AS, Deshmukh S, et al · · 2019 · cited 162× · PMID 31085178 · DOI 10.1016/j.ccell.2019.04.004
Therapeutic strategies for diffuse midline glioma from high-throughput combination drug screening.
Lin GL, Wilson KM, Ceribelli M, Stanton BZ, et al · · 2019 · cited 158× · PMID 31748226 · DOI 10.1126/scitranslmed.aaw0064
Developmental origins and emerging therapeutic opportunities for childhood cancer.
Filbin M, Monje M. · · 2019 · cited 150× · PMID 30842674 · DOI 10.1038/s41591-019-0383-9
Pre-Clinical Study of Panobinostat in Xenograft and Genetically Engineered Murine Diffuse Intrinsic Pontine Glioma Models.
Hennika T, Hu G, Olaciregui NG, Barton KL, et al · · 2017 · cited 124× · PMID 28052119 · DOI 10.1371/journal.pone.0169485
Recent developments in epigenetic cancer therapeutics: clinical advancement and emerging trends.
Nepali K, Liou JP. · · 2021 · cited 122× · PMID 33840388 · DOI 10.1186/s12929-021-00721-x
Spatially controlled construction of assembloids using bioprinting.
Roth JG, Brunel LG, Huang MS, Liu Y, et al · · 2023 · cited 83× · PMID 37468483 · DOI 10.1038/s41467-023-40006-5
Diffuse Intrinsic Pontine Glioma: From Diagnosis to Next-Generation Clinical Trials.
Vitanza NA, Monje M. · · 2019 · cited 81× · PMID 31290035 · DOI 10.1007/s11940-019-0577-y

Verify or expand the search:

Other trials of LBH589

Trials testing the same drug.

NCT00532675 — Safety Study of LBH589 When Given in Combination With Lenalidomide and Dexamethasone in Adult Patients With Multiple Mye · Phase 1 · completed
NCT00445068 — Efficacy and Safety of LBH589B in Adult Patients With Multiple Myeloma · Phase 2 · terminated
NCT00449761 — Efficacy and Safety of LBH589B in Adult Patients With Refractory Chronic Myeloid Leukemia in Accelerated or Blast Phase · Phase 2 · terminated
NCT00451035 — Efficacy and Safety of LBH589 in Adult Patients With Refractory Chronic Myeloid Leukemia (CML) in Chronic Phase · Phase 2 · terminated
NCT00621244 — A Study of Oral LBH589 in Adult Patients With Advanced Hematological Malignancies · Phase 1, PHASE2 · completed

Other recruiting trials for Glioma

Currently open trials in the same condition.

NCT07486713 — Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies · Phase 4 · recruiting
NCT07236840 — Self-administered Remote Neurological Examination Using Mobile Application in Patients With Brain Tumors · recruiting
NCT07450872 — Symptom Cluster Heterogeneity and Gut Microbiota Mechanisms in Childhood Cancer Survivors · recruiting
NCT07405372 — Construction and Application of an IDH Mutation-Targeted PET/MRI Imaging Framework for Precision Diagnosis of Gliomas · recruiting
NCT07338539 — BIO-SHORT: Biologically Guided Short-Course Hypofractionated RT for Poor-Prognosis GBM · Phase 2 · recruiting

Other Pediatric Brain Tumor Consortium trials

Trials by the same sponsor.

NCT04482933 — HSV G207 With a Single Radiation Dose in Children With Recurrent High-Grade Glioma · Phase 2 · withdrawn
NCT05934630 — Testing Cerebrospinal Fluid for Cell-free Tumor DNA in Children, Adolescents, and Young Adults With Brain Tumors · suspended
NCT04743661 — 131I-Omburtamab, in Recurrent Medulloblastoma and Ependymoma · Phase 2 · active not recruiting
NCT04295759 — INCB7839 in Treating Children With Recurrent/Progressive High-Grade Gliomas · Phase 1 · completed
NCT04201457 — A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration · Phase 1, PHASE2 · active not recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT02717455 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Pediatric Brain Tumor Consortium
Last refreshed: 25 April 2024

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02717455.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing