A Phase 2 interventional study of Imetelstat Sodium and Laboratory Biomarker Analysis in Hepatoblastoma, Previously Treated Childhood Rhabdomyosarcoma and Recurrent Childhood Liver Cancer, sponsored by Children's Oncology Group. Withdrawn. Open to participants aged 1 Year to 30 Years. Per ClinicalTrials.gov, last updated 2018-08-07.
Sponsored by Children's Oncology Group · Phase 2, Interventional, and Treatment
This phase II trial studies the side effects and how well imetelstat sodium works in treating younger patients with relapsed or refractory solid tumors. Imetelstat sodium may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.
PRIMARY OBJECTIVES:
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.
SECONDARY OBJECTIVES:
I. To determine the time to progression following treatment with imetelstat in children with relapsed or refractory solid tumors.
TERTIARY OBJECTIVES:
I. To measure tumor telomere length in archival samples, and to correlate telomere length to the clinical outcome of the study.
OUTLINE:
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.
Patients with any of the following tumors who have relapsed or refractory disease are eligible:
Patients must have radiographically measurable disease (with the exception of neuroblastoma)
Note: the following do not qualify as measurable disease:
For patients with solid tumors without bone marrow involvement:
For patients with solid tumors without bone marrow involvement:
For patients with solid tumors without bone marrow involvement:
For patients with solid tumors and known bone marrow metastatic disease:
For patients with solid tumors and known bone marrow metastatic disease:
For patients with solid tumors and known bone marrow metastatic disease:
Hemoglobin >= 8.0 g/dL
Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:
Exclusion Criteria:
patients who are receiving cyclosporine, tacrolimus or other agents to prevent either graft-versus-host disease post bone marrow transplant or organ rejection post-transplant are not eligible for this trial
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: Laboratory Biomarker Analysis
Given IV
Also known as: GRN163L, GRN163L, Sodium Salt
Optional correlative studies
Objective response (complete response [CR] or partial response [PR]) according to Response Evaluation Criteria in Solid Tumors (RECIST) criteria or MIBG scoring criteria
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.
Time frame: Up to 126 days (6 courses)
Incidence of grade 3 or higher adverse events using the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0
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.
Time frame: Up to 126 days (6 courses)
Progression-free survival
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.
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
Telomerase length by quantitative polymerase chain reaction
The relationship between tumor telomere length and probability of response (as CR or PR) with imetelstat sodium will be assessed using logistic regression.
Time frame: Baseline
No study locations are listed for this record.
This study is withdrawn, as verified in May 2014. You cannot join it, but the record below documents what was studied.
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Children's Oncology Group