Clinical Trial: Imetelstat Sodium in Treating Younger Patients With Relapsed or Refractory Solid Tumors

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

Official Title: A Phase II Study of Imetelstat (GRN163L, NSC# 754228) in Children With Relapsed or Refractory Solid Tumors

Brief Summary: 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.

Detailed Summary:

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.

Current Primary Outcome:

• 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) ]
  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) ]
  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.

Original Primary Outcome:

• 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) ]
  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) ]
  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 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.

Current Secondary Outcome: 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 ]

Original Secondary Outcome: Same as current

Information By: Children's Oncology Group

Dates:
Date Received: December 10, 2013
Date Started: June 2014
Date Completion:
Last Updated: February 8, 2017
Last Verified: February 2017