A Study of Panobinostat in Pediatric Patients With Solid Tumors Including MRT/ATRT
Last updated on July 2021Recruitment
- Recruitment Status
- Active, not recruiting
- Estimated Enrollment
- Same as current
Summary
- Conditions
- Atypical Teratoid/Rhabdoid Tumor
- Malignant Rhabdoid Tumor
- Recurrent Brain Tumor, Childhood
- Rhabdoid Tumor
- Type
- Interventional
- Phase
- Phase 2
- Design
- Allocation: Non-RandomizedIntervention Model: Parallel AssignmentMasking: None (Open Label)Primary Purpose: Treatment
Participation Requirements
- Age
- Younger than 39 years
- Gender
- Both males and females
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)...
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.
Tracking Information
- NCT #
- NCT04897880
- Collaborators
- National Health and Medical Research Council, Australia
- Secura Bio, Inc.
- Investigators
- Not Provided