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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04897880




Registration number
NCT04897880
Ethics application status
Date submitted
6/05/2021
Date registered
24/05/2021
Date last updated
8/06/2022

Titles & IDs
Public title
A Study of Panobinostat in Pediatric Patients With Solid Tumors Including MRT/ATRT
Scientific 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
Secondary ID [1] 0 0
ACTRN12618000321246
Secondary ID [2] 0 0
ACCT008/ASSG35
Universal Trial Number (UTN)
Trial acronym
NORTH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rhabdoid Tumor 0 0
Atypical Teratoid/Rhabdoid Tumor 0 0
Malignant Rhabdoid Tumor 0 0
Recurrent Brain Tumor, Childhood 0 0
Condition category
Condition code
Cancer 0 0 0 0
Brain
Cancer 0 0 0 0
Kidney
Cancer 0 0 0 0
Children's - Other

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Panobinostat

Experimental: Osteosarcoma [arm closed] -

Experimental: Malignant Rhabdoid Tumor/Atypical Teratoid Rhabdoid Tumor -

Experimental: Neuroblastoma [arm closed] -


Treatment: Drugs: Panobinostat
Panobinostat capsules, 10mg, starting at a de-escalated dose of 8mg/m2 per day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Efficacy as measured by Clinical Benefit Rate (percentage of patients with stable disease or better using MRI/CT imaging)
Timepoint [1] 0 0
4 months after intervention commencement
Primary outcome [2] 0 0
Safety, as assessed by incidence of adverse events graded according to the NCI-CTCAE, version 4.0
Timepoint [2] 0 0
1 week to 12 months after intervention commencement
Secondary outcome [1] 0 0
Clinical Benefit Rate: Percentage of patients with stable disease or better using functional imaging (MIBG or FDG-PET).
Timepoint [1] 0 0
Every 2 months for 12 months after treatment commencement
Secondary outcome [2] 0 0
Time to progression calculated as the time from registration to date of event defined as the first documented progression or death resulting from underlying cancer.
Timepoint [2] 0 0
2 years after completion of treatment
Secondary outcome [3] 0 0
Overall Survival calculated as the time from registration to date of death
Timepoint [3] 0 0
2 years after completion of treatment

Eligibility
Key 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.
Minimum age
No limit
Maximum age
39 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/01/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/12/2021
Sample size
Target
Accrual to date
Final
25
Recruitment in Australia
Recruitment state(s)
NSW,SA,TAS,VIC,WA
Recruitment hospital [1] 0 0
John Hunter Children's Hospital - New Lambton
Recruitment hospital [2] 0 0
Sydney Children's Hospital - Randwick
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [4] 0 0
Women's and Children's Hospital - North Adelaide
Recruitment hospital [5] 0 0
Royal Hobart Hospital - Hobart
Recruitment hospital [6] 0 0
Monash Children's Hospital - Clayton
Recruitment hospital [7] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [8] 0 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
2305 - New Lambton
Recruitment postcode(s) [2] 0 0
2031 - Randwick
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment postcode(s) [4] 0 0
5006 - North Adelaide
Recruitment postcode(s) [5] 0 0
7000 - Hobart
Recruitment postcode(s) [6] 0 0
3168 - Clayton
Recruitment postcode(s) [7] 0 0
3052 - Parkville
Recruitment postcode(s) [8] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
North Carolina
Country [2] 0 0
New Zealand
State/province [2] 0 0
Auckland
Country [3] 0 0
New Zealand
State/province [3] 0 0
Christchurch

Funding & Sponsors
Primary sponsor type
Other
Name
Australian & New Zealand Children's Haematology/Oncology Group
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
National Health and Medical Research Council, Australia
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Commercial sector/Industry
Name [2] 0 0
Secura Bio, Inc.
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04897880
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04897880