Rhabdoid TumorPhase 2NORTHNCT04897880
A Study of Panobinostat in Pediatric Patients With Solid Tumors Including MRT/ATRT
A Phase 2 study testing Panobinostat in people with Rhabdoid Tumor and Atypical Teratoid/Rhabdoid Tumor, run by Australian & New Zealand Children's Haematology/Oncology Group at 11 sites in 3 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 39 years
- Conditions the study is for
- Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor
- What is being tested?
- Panobinostat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 11 sitesin 3 countries
- Study dates
- 9 Jan 2019to 8 May 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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 locations and contacts
11 sites in 3 countries.
11 sites in 3 countries
Australia8 sites
Monash Children's Hospital
Clayton, Victoria, 3168
Royal Hobart Hospital
Hobart, Tasmania, 7000
Perth Children's Hospital
Nedlands, Western Australia, 6009
John Hunter Children's Hospital
New Lambton, New South Wales, 2305
Women's and Children's Hospital
North Adelaide, South Australia, 5006
The Royal Children's Hospital
Parkville, Victoria, 3052
Sydney Children's Hospital
Randwick, New South Wales, 2031
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
New Zealand2 sites
Christchurch Hospital
Christchurch, 8011
Starship Children's Hospital
Grafton, Auckland, 1023
United States1 site
The Preston Robert Tisch Brain Tumor Center, Duke University Medical Center
Durham, North Carolina, 27710
About this study
Official title: A Phase II Study of Panobinostat in Pediatric, Adolescent and Young Adult Patients With Solid Tumors Including Osteosarcoma, Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumors and Neuroblastoma
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.
Read the detailed description
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.
- Conditions
- Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor, Recurrent Brain Tumor, Childhood
- Being tested
- Panobinostat
- Keywords
- Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor, Recurrent Brain Tumor, Childhood, Ashley, Pro00107447
- Medical subject headings
- Rhabdoid Tumor, Brain Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PanobinostatDrug
Also called Farydak®
Panobinostat capsules, 10mg, starting at a de-escalated dose of 8mg/m2 per day
| Study group | Role | What they receive |
|---|---|---|
| Osteosarcoma [arm closed] | Experimental | Not described Receives: Drug: Panobinostat |
| Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumor | Experimental | Not described Receives: Drug: Panobinostat |
| Neuroblastoma [arm closed] | Experimental | Not described Receives: Drug: Panobinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event free survival
Estimated 2-year Event free survival (EFS). EFS is calculated as the time from study enrolment to first documented disease progression, relapse or second malignancy, or death from any cause.
Measured: Up to 2 years after study enrolment
Overall Survival
Estimated 2-year Overall Survival (OS). OS is calculated as the time from study enrolment to death from any cause.
Measured: Up to 2 years after study enrolment
Safety: Adverse events summarised by grade and type
Graded and defined by CTCAE Version 4
Measured: From 1 week to 12 months after intervention commencement
Secondary outcomes (1)
Efficacy as measured by Clinical Benefit Rate
Clinical benefit rate as the percentage of patients with stable disease or better using MRI/CT imaging).
Stable disease is defined as MRT/ATRT/Osteosarcoma with CR/PR/MR/SD/ overall response.
Neuroblastoma with CR/PR/SD or Non-CR/Non-PD overall response.
Measured: At 6 and 12 months after intervention commencement
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2021
- Study start9 Jan 2019
- Primary completion8 May 2024
- Study completion8 May 2024
Why it stopped: Drug supply
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rhabdoid TumorStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- Australian & New Zealand Children's Haematology/Oncology Group
- Sponsor type
- University, hospital or other
- Collaborators
- National Health and Medical Research Council, Australia, Secura Bio, Inc.
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04897880
- Sponsor's protocol ID
- ACCT008/ASSG35
- Other IDs
- ACTRN12618000321246
- Acronym
- NORTH
- Status last verified
- Feb 2025
- First submitted
- 6 May 2021
- Last updated
- 24 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Panobinostat in Pediatric, Adolescent and Young Adult Patients With Solid Tumors Including Osteosarcoma, Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumors and Neuroblastoma
- ClinicalTrials.gov ID
- NCT04897880
- Acronym / sponsor ID
- NORTH / ACCT008/ASSG35
- Conditions
- Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor, Recurrent Brain Tumor, Childhood
- Keywords
- Rhabdoid Tumor, Atypical Teratoid/Rhabdoid Tumor, Malignant Rhabdoid Tumor, Recurrent Brain Tumor, Childhood, Ashley, Pro00107447
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Drug supply
- Enrolment
- 25 participants (actual)
- Lead sponsor
- Australian & New Zealand Children's Haematology/Oncology GroupUniversity, hospital or other
- Collaborators
- National Health and Medical Research Council, Australia, Secura Bio, Inc.
- Study start
- 9 Jan 2019 (actual)
- Primary completion
- 8 May 2024 (actual)
- Study completion
- 8 May 2024 (actual)
- First posted
- 24 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Osteosarcoma [arm closed] | Experimental | Drug: Panobinostat |
| Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumor | Experimental | Drug: Panobinostat |
| Neuroblastoma [arm closed] | Experimental | Drug: Panobinostat |
Who may qualify
- Ages
- Up to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (18 listed)
- 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.
Key exclusion criteria (13 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PanobinostatAlso: Farydak®Panobinostat capsules, 10mg, starting at a de-escalated dose of 8mg/m2 per day | Drug | Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumor, Neuroblastoma [arm closed], Osteosarcoma [arm closed] |
Primary outcome
Event free survival
Time frame: Up to 2 years after study enrolment
Estimated 2-year Event free survival (EFS). EFS is calculated as the time from study enrolment to first documented disease progression, relapse or second malignancy, or death from any cause.
Overall Survival
Time frame: Up to 2 years after study enrolment
Estimated 2-year Overall Survival (OS). OS is calculated as the time from study enrolment to death from any cause.
Safety: Adverse events summarised by grade and type
Time frame: From 1 week to 12 months after intervention commencement
Graded and defined by CTCAE Version 4
1 secondary outcome
Efficacy as measured by Clinical Benefit Rate
Time frame: At 6 and 12 months after intervention commencement
Results and safety
The study was stopped early (reason given: Drug supply). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Panobinostat in people with Rhabdoid Tumor and Atypical Teratoid/Rhabdoid Tumor, run by Australian & New Zealand Children's Haematology/Oncology Group at 11 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Australia, New Zealand, United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Drug supply). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Event free survival (time frame: Up to 2 years after study enrolment). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2025.