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

NCT02011126 | Withdrawn Phase 2

• 3 other identifiers: ADVL1321NCI-2013-01640U10CA098543
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Hepatoblastoma; Previously Treated Childhood Rhabdomyosarcoma; Recurrent Childhood Liver Cancer; Recurrent Childhood Rhabdomyosarcoma; Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor; Recurrent Neuroblastoma; Recurrent Osteosarcoma; Rhabdomyosarcoma

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Up to 126 days (6 courses)

Secondary Outcomes (1)

• Progression-free survival

  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

Other Outcomes (1)

• Telomerase length by quantitative polymerase chain reaction

  Baseline

Study Arms (1)

Treatment (imetelstat sodium)

EXPERIMENTAL

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

Interventions

Imetelstat Sodium DRUG

Given IV

Also known as: GRN163L, GRN163L, Sodium Salt

Treatment (imetelstat sodium)

Laboratory Biomarker Analysis OTHER

Optional correlative studies

Treatment (imetelstat sodium)

Eligibility Criteria

• Age: 1 Year - 30 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patients with any of the following tumors who have relapsed or refractory disease are eligible:
• Osteosarcoma
• Ewing?s sarcoma / peripheral primitive neuroectodermal tumor (PNET)
• Rhabdomyosarcoma
• Neuroblastoma (measurable or evaluable disease)
• Hepatoblastoma
• Patients must have had histologic verification of malignancy at original diagnosis or relapse
• Patients must have radiographically measurable disease (with the exception of neuroblastoma)
• Measurable disease is defined as the presence of at least one lesion on magnetic resonance imaging (MRI) or computed tomography (CT) scan that can be accurately measured with the longest diameter a minimum of 10 mm in at least one dimension (CT scan slice thickness no greater than 5 mm)
• Note: the following do not qualify as measurable disease:
• Malignant fluid collections (e.g., ascites, pleural effusions)
• Bone marrow infiltration
• Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography \[PET\] scans) except as defined below for neuroblastoma
• Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
• Previously radiated lesions that have not demonstrated clear progression post radiation

You may not qualify if:

• Patients who are pregnant or breast-feeding are not eligible for this study; negative pregnancy tests must be obtained in girls who are post-menarchal; males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method for the duration of study therapy; study drug may also potentially be secreted in milk and therefore breastfeeding women are excluded
• Growth factors that support platelet or white cell number or function must not have been administered within the 7 days prior to enrollment (14 days if pegfilgrastim)
• Patients requiring corticosteroids who have not been on a stable or decreasing dose of corticosteroid for the 7 days prior to enrollment are not eligible
• Patients who are currently receiving another investigational drug are not eligible
• Patients who are currently receiving other anti-cancer agents are not eligible
• Anti-graft-versus-host disease (GVHD) or agents to prevent organ rejection post-transplant:
• 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 who have an uncontrolled infection are not eligible
• Patients who have received prior treatment with imetelstat are not eligible
• Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
• Patients with prior allogeneic transplants are not eligible

Sponsors & Collaborators

• Children's Oncology Group: lead
• National Cancer Institute (NCI): collaborator

MeSH Terms

Conditions

Hepatoblastoma; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma; Osteosarcoma; Rhabdomyosarcoma

Interventions

imetelstat; GRN163L peptide

Condition Hierarchy (Ancestors)

Neoplasms, Complex and Mixed; Neoplasms by Histologic Type; Neoplasms; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue; Neoplasms, Bone Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Sarcoma; Myosarcoma; Neoplasms, Muscle Tissue

Study Officials

• Patrick Thompson

  Children's Oncology Group

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 10, 2013
• First Posted: December 13, 2013
• Study Start: June 30, 2014
• Primary Completion: March 25, 2016
• Last Updated: August 7, 2018

Record last verified: 2014-05