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

• Status: Recruiting
• Trial end date: 2025-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Australian & New Zealand Children's Haematology/Oncology Group
• Collaborators: National Health and Medical Research Council, Australia; Secura Bio; Secura Bio, Inc.
• Treatments: Panobinostat

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.