Imetelstat Sodium in Treating Younger Patients With Relapsed or Refractory Solid Tumors
Previously Treated Childhood Rhabdomyosarcoma
Recurrent Childhood Liver Cancer
Recurrent Childhood Rhabdomyosarcoma
Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor
Drug: imetelstat sodium
Other: laboratory biomarker analysis
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase II Study of Imetelstat (GRN163L, NSC# 754228) in Children With Relapsed or Refractory Solid Tumors|
- Objective response (complete response [CR] or partial response [PR]) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria or MIBG scoring criteria [ Time Frame: Up to 126 days (6 courses) ] [ Designated as safety issue: No ]Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed accounting for the two-stage design. Each disease stratum will be reported separately.
- Incidence of grade 3 or higher adverse events using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: Up to 126 days (6 courses) ] [ Designated as safety issue: Yes ]Toxicity tables will be constructed to summarize the observed incidence in each reporting period by type of toxicity and grade. The relative frequency of each type of toxicity will be quantified as the number of toxicity-evaluable cycles in which the adverse event (AE) was noted at grade 3 or higher considered by the treating physician to be possibly, probably or definitely related to one of the agents in the regimen divided by the number of toxicity-evaluable courses administered to patients enrolled on the trial.
- Progression-free survival [ Time Frame: Date of enrollment until the end progression-free interval (PFI) date, calculated as the date of disease progression, date of death, date of removal of all tumor by surgery or last patient contact, whichever occurs first, assessed up to 3 years ] [ Designated as safety issue: No ]The probability of remaining progression-free as a function of days since enrollment will be calculated according to the method of Gray accounting for censoring and the competing events.
- Telomerase length by quantitative polymerase chain reaction [ Time Frame: Baseline ] [ Designated as safety issue: No ]The relationship between tumor telomere length and probability of response (as CR or PR) with imetelstat sodium will be assessed using logistic regression.
|Study Start Date:||June 2014|
|Estimated Primary Completion Date:||March 2016 (Final data collection date for primary outcome measure)|
Experimental: Treatment (imetelstat sodium)
Patients receive imetelstat sodium IV over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity.
Drug: imetelstat sodium
Other Names:Other: laboratory biomarker analysis
Optional correlative studies
I. To determine the objective response rate, defined as partial response or better, of imetelstat (imetelstat sodium) in children with relapsed or refractory solid tumors.
II. To further define and describe the toxicities associated with imetelstat in children with recurrent/refractory solid tumors.
I. To determine the time to progression following treatment with imetelstat in children with relapsed or refractory solid tumors.
I. To measure tumor telomere length in archival samples, and to correlate telomere length to the clinical outcome of the study.
Patients receive imetelstat sodium intravenously (IV) over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 36 courses in the absence of disease progression or unacceptable toxicity.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02011126
|Principal Investigator:||Patrick Thompson||Children's Oncology Group|