A Phase I Study of SB939 in Pediatric Patients With Refractory Solid Tumours and Leukemia
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase I Study of SB939 in Pediatric Patients With Refractory Solid Tumours and Leukemia|
- Part A: Maximum Tolerated Dose and RP2D in solid tumours [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Part A: patients must have recurrent or refractory solid tumours, lymphoma or CNS tumours (excluding diffuse intrinsic pontine gliomas)
Purpose is to determine recommended phase II dose (RP2D) of oral SB939 in pediatric patients with solid tumours, with SB939 administered at a starting dose of 25 mg/m2 (70% of the adult recommended phase II dose), and given orally every other day three times / week (e.g. Monday / Wednesday /Friday OR Tuesday / Thursday / Saturday) for three consecutive weeks, followed by one week off-dosing.
- Part B: Tolerability [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Part B: patients must have recurrent or refractory leukemia
Tolerability of the solid tumour RP2D in patients with recurrent or refractory leukemia once the RP2D has been established in solid tumours.
- Part C: Recommended Phase 2 Dose (RP2D) and Tolerability [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]
Part C: patients must have neuroblastoma, or medulloblastoma/CNS primitive neuroectodermal tumour (PNET)
RP2D of oral SB939 in combination with a fixed dose of 13-cisretinoic acid in children with refractory or recurrent neuroblastoma, medulloblastoma / CNS neuroectodermal tumour (PNET), using the recommended dose determined in Part A of this study.
- Pharmacokinetics [ Time Frame: 24 months ] [ Designated as safety issue: No ]characterize the pharmacokinetics of SB939 in a pediatric population
- Anti-tumour activity [ Time Frame: 24 months ] [ Designated as safety issue: No ]antitumour activity of SB939 in pediatric tumours when given as a single agent, and when given in combination with 13-cis-retinoic acid.
|Study Start Date:||September 2010|
|Study Completion Date:||January 2014|
|Primary Completion Date:||January 2012 (Final data collection date for primary outcome measure)|
Dose Levels for Part A
-1 - 20mg/m2 - oral - Every other day three times/week1 for three consecutive weeks, followed by one week off-dosing
4+ - Previous level + 10mg/m2 - oral - Every other day three times/week1 for three consecutive weeks, followed by one week off-dosing
In Part A of this study, SB939 was given to children with solid tumours. The purpose of Part A of this study is to ind the highest dose of a new drug SB939 that can be giben to children without causing very severe side effects that are tolerable.
In Part B of this study, SB939 will be given to children with leukemia. The purpose of Part B, is to see whether the dose that was determined to be the best dose for patients with solid tumours is also the best dose for children with leukemia.
In Part C of this study, SB939 will be given together with 13-cis-retinoic acid. The purpose of Part C, is to see whether the SB939 dose that was determined to be the best dose in Part A is also the best dose when given in combination with 13-cis-retinoic acid.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01184274
|Alberta Children's Hospital|
|Calgary, Alberta, Canada, T3B 6A8|
|Stollery Children's Hospital|
|Edmonton, Alberta, Canada, T6G 2B7|
|Canada, British Columbia|
|Children's and Women's Health Centre of BC Branch|
|Vancouver, British Columbia, Canada, V6H 3V4|
|Winnipeg, Manitoba, Canada, R3E 0V9|
|Canada, Nova Scotia|
|Izaak Walton Killam (IWK) Health Centre|
|Halifax, Nova Scotia, Canada, B3K 6R8|
|Children's Hospital of Eastern Ontario|
|Ottawa, Ontario, Canada, K1H 8L1|
|Hospital for Sick Children|
|Toronto, Ontario, Canada, M5G 1X8|
|Montreal, Quebec, Canada, H3T 1C5|
|Study Chair:||Sylvain Baruchel||Hospital for Sick Children, Toronto Ontario Canada|