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A Study of Panobinostat in Pediatric Patients With Solid Tumors Including MRT/ATRT

Study Purpose

This trial is evaluating the anti-tumor activity and side effects of panobinostat in treating patients with osteosarcoma, malignant rhabdoid tumor/atypical teratoid rhabdoid tumor (MRT/ATRT), and neuroblastoma.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.

An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.

Searching Both is inclusive of interventional and observational studies.

Eligible Ages N/A - 39 Years
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - Patients must be < 40 years of age.
  • - Patient must have been histologically diagnosed with osteosarcoma, neuroblastoma or MRT/ATRT at time of diagnosis or relapse.
[osteosarcoma and neuroblastoma arms are closed to recruitment].
  • - Patient disease is refractory to conventional therapy, in the case of osteosarcoma, neuroblastoma and MRT/ATRT or there is an absence of effective conventional therapy available in the case of ATRT.
Patients must have stable disease (SD) or better following treatment with salvage therapy.
  • - Karnofsky performance level greater than or equal to 60% for patients 16 years of age and greater, OR Lansky performance levels greater than or equal to 60% for patients less than 16 years of age.
  • - Life expectancy of greater than 8 weeks.
  • - Fully recovered from acute toxic effects of all prior chemotherapy, immunotherapy or radiotherapy prior to entering study.
  • - Patients with CNS tumours who are receiving dexamethasone are on a stable/decreasing dose for at least 1 week.
  • - Adequate BM function.
  • - Adequate renal function.
  • - Adequate liver function.
  • - Adequate cardiac function.
  • - Adequate pulmonary function.
  • - Adequate CNS function - seizure free for at least 2 months.
  • - Adequate serum calcium, magnesium and potassium concentrations.
  • - If female and post-menarchal, pregnancy test must be negative.
  • - If of reproductive potential, have agreed to use effective contraceptive method.
  • - If female and lactating, have agreed not to breastfeed.
  • - Patient and/or their legal guardian have signed a written informed consent form.

Exclusion Criteria:

  • - Have received myelosuppressive chemotherapy and/or biologic therapy within 3 weeks (4 weeks if prior nitrosourea).
  • - Have received local palliative radiotherapy within 2 weeks.
  • - Have received craniospinal radiotherapy within 3 weeks.
  • - Have received greater than or equal to 50% radiation of the pelvis within 6 weeks.
  • - Have received other substantial BM radiation within 6 weeks.
  • - Have received growth factor(s) within 1 week.
  • - Are receiving enzyme inducing anticonvulsant therapy.
  • - Are receiving medications associated with prolongation of QTc interval.
  • - Are receiving hydrochlorothiazide.
  • - Are receiving metronidazole and/or disulfiram.
  • - Have uncontrolled sepsis.
  • - Have previously received panobinostat.
  • - Have symptoms of congestive heart failure, uncontrolled cardiac rhythm disturbance, or a QTc greater than or equal to 450msec.

Trial Details

Trial ID:

This trial id was obtained from, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.


Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Australian & New Zealand Children's Haematology/Oncology Group
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Overall Status Recruiting
Countries Australia, New Zealand, United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor, Recurrent Brain Tumor, Childhood
Additional Details

This is an open label, phase II, multi-centre study evaluating the anti-tumor activity of continuous, low dose of panobinostat in patients with recurrent or refractory solid tumors stratified by primary histology into osteosarcoma, malignant rhabdoid tumor/atypical teratoid rhabdoid tumor (MRT/ATRT), and neuroblastoma. Patients will be stratified at study entry by tumor type into three strata: osteosarcoma, MRT/ATRT and neuroblastoma [osteosarcoma and neuroblastoma arms are closed to enrolment]. Patients will be enrolled onto the study following completion of their conventional therapy including chemotherapy and/or radiation treatment and completion of a three-week wash out period. Panobinostat will then be administered as a continuous oral dose (starting at a de-escalated dose of 8mg/m2 per day), for up to 12 courses, a total of 48 weeks. The minimum dose is 2mg/m2 per day. Dosing will follow a dose de-escalation or escalation scheme for each stratum which will be determined by biological effect of the drug (measured in patient peripheral blood samples) and levels of toxicity (measured by dose limiting toxicity and adverse events observed). Dose levels for subsequent enrolments in each strata will be based on the de-escalated or escalated dose in each cohort. The final dose per strata will be that which achieves significant biological effect with acceptable toxicity that is maintained for a 4 week period. Patients or their parents/guardians will be required to maintain a drug diary to monitor drug usage throughout the trial. Patients will be followed for up to 2 years from completion of study therapy.

Arms & Interventions


Experimental: Osteosarcoma [arm closed]

Experimental: Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumor

Experimental: Neuroblastoma [arm closed]


Drug: - Panobinostat

Panobinostat capsules, 10mg, starting at a de-escalated dose of 8mg/m2 per day

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Durham, North Carolina




The Preston Robert Tisch Brain Tumor Center, Duke University Medical Center

Durham, North Carolina, 27710

International Sites

John Hunter Children's Hospital, New Lambton, New South Wales, Australia


Active, not recruiting


John Hunter Children's Hospital

New Lambton, New South Wales, 2305

Sydney Children's Hospital, Randwick, New South Wales, Australia


Active, not recruiting


Sydney Children's Hospital

Randwick, New South Wales, 2031

The Children's Hospital at Westmead, Westmead, New South Wales, Australia


Active, not recruiting


The Children's Hospital at Westmead

Westmead, New South Wales, 2145

Women's and Children's Hospital, North Adelaide, South Australia, Australia


Active, not recruiting


Women's and Children's Hospital

North Adelaide, South Australia, 5006

Royal Hobart Hospital, Hobart, Tasmania, Australia


Active, not recruiting


Royal Hobart Hospital

Hobart, Tasmania, 7000

Monash Children's Hospital, Clayton, Victoria, Australia


Active, not recruiting


Monash Children's Hospital

Clayton, Victoria, 3168

The Royal Children's Hospital, Parkville, Victoria, Australia


Active, not recruiting


The Royal Children's Hospital

Parkville, Victoria, 3052

Perth Children's Hospital, Nedlands, Western Australia, Australia


Active, not recruiting


Perth Children's Hospital

Nedlands, Western Australia, 6009

Starship Children's Hospital, Grafton, Auckland, New Zealand


Active, not recruiting


Starship Children's Hospital

Grafton, Auckland, 1023

Christchurch Hospital, Christchurch, New Zealand


Active, not recruiting


Christchurch Hospital

Christchurch, , 8011