Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors

Stopped early · Phase 2

Conditions studied: Anaplastic Astrocytoma, Anaplastic Ependymoma, Astrocytoma, Grade II, Ependymoma, Giant Cell Glioblastoma, Glioblastoma, Gliosarcoma, Oligodendroglioma, Brainstem Tumors

In brief

This molecular biology and phase II trial studies how well imetelstat sodium works in treating younger patients with recurrent or refractory brain tumors. Imetelstat sodium may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Key facts

Study ID: NCT01836549
Run by: Pediatric Brain Tumor Consortium
People needed: 43
Starts: 2013-03-01
Expected to finish: 2016-04-01
Last updated by the study team: 2018-07-20

Who can join

Age: 1 and older, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

MOLECULAR BIOLOGY STUDY
Tumor: Histologically confirmed Dx of ependymoma or HGG (such as anaplastic astrocytoma, glioblastoma, gliosarcoma, or anaplastic oligodendroglioma) that is recurrent or refractory to conventional therapy.
Subjects must have clinical indications for surgical resection and be amenable to receiving imetelstat prior to tumor resection. Subjects who require emergent surgery are not eligible for the Molecular Biology study.
Subjects must provide, fresh flash frozen tumor samples (target 50 mg tissue; as low as 20 mg is adequate) from the time of diagnosis or previous recurrence for the assessment of tumor telomerase activity by the TRAP assay.
PHASE II STUDY
Tumor: Subjects must have recurrent or refractory disease with a histological Dx from either the initial presentation or at the time of recurrence. The requirement for histologic verification is waived for subjects with DIPG (stratum D). The following diagnoses are eligible and will be treated in separate strata (B-D): (B) recurrent or refractory high-grade glioma, (such as anaplastic astrocytoma, glioblastoma multiforme, gliosarcoma, anaplastic oligodendroglioma); (C) recurrent or refractory ependymoma; (D) recurrent or refractory DIPG (diagnosis by imaging characteristics acceptable; no histologic confirmation required)
Slides from either initial Dx or relapse must be available for central pathology review for Strata B-C. Tissue slides must be sent per Section 10.1. If tissue slides are unavailable, the study chair must be notified prior to study enrollment.
All subjects must have bi-dimensionally measurable disease in the brain and/or spine, defined as at least one lesion that can be accurately measured in at least two planes in order to be eligible for this study. Subjects who are enrolled on the Molecular Biology trial and who have measurable disease after the surgical resection and meet all other eligibility criteria for the Phase II study will be counted towards the accrual of the Phase II study.
FOR BOTH MOLECULAR BIOLOGY AND PHASE II STUDIES
Subjects with neurological deficits should have deficits that are stable for a minimum of 1 week prior to registration; a baseline detailed neurological exam should clearly document the neurological status of the subject at the time of registration on the study
Karnofsky >= 50% for > 16 years of age; Lansky >= 50% for children < 16 years of age documented within 14 days of study registration and within 7 days of the start of study drug administration
Hemoglobin >= 8 g/dL (may receive blood transfusions)
Absolute neutrophil count > 1,000/ul
Platelet count >= 100,000/ul (transfusion independent defined as no platelet transfusions with a 4 week period prior to enrollment)
Serum bilirubin < 2.0 mg/dL (patients with Gilbert syndrome, serum bilirubin < 3.0 x upper limit of normal [ULN])
Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT]) =< 3 x institutional ULN
Alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3 x institutional ULN
Alkaline phosphatase < 2.5 x institutional ULN
Albumin >= 2 g/dL
Adequate coagulation defined as activated partial thromboplastin time (aPTT) < 1.2 x ULN
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:
Age 1 to < 2 years: maximum serum creatinine (mg/mL) 0.6 for males and 0.6 for females
Age 2 to < 6 years: maximum serum creatinine (mg/mL) 0.8 for males and 0.8 for females
Age 6 to < 10 years: maximum serum creatinine (mg/mL) 1 for males and 1 for females
Age 10 to < 13 years: maximum serum creatinine (mg/mL) 1.2 for males and 1.2 for females

You may not qualify if…

Subjects must not be receiving any other investigational agents
Subjects with inability to return for follow-up visits or obtain follow-up studies required to assess toxicity to therapy
History of allergic reactions attributed to compounds of similar chemical or biologic composition to imetelstat
Known coagulopathy or bleeding diathesis
Subjects with imaging evidence of CNS hemorrhage on baseline MRI obtained within 14 days prior to study enrollment are not eligible; Note: The presence of small punctate areas consistent with hemorrhage will not exclude subjects from participation
Use of systemic anticoagulant medications
Uncontrolled intercurrent illness including, but not limited to, ongoing or active serious infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, cirrhosis or psychiatric illness/social situations that would limit compliance with study requirements

Where it is running

Childrens Hospital Los Angeles — Los Angeles, California, United States
Lucile Packard Children's Hospital at Stanford University Medical Center — Palo Alto, California, United States
Children's National Medical Center — Washington D.C., District of Columbia, United States
Lurie Children's Hospital- Chicago — Chicago, Illinois, United States
NCI - Pediatric Oncology Branch — Bethesda, Maryland, United States
Memorial Sloan-Kettering Cancer Center — New York, New York, United States
Duke Comprehensive Cancer Center — Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
Saint Jude Children's Research Hospital — Memphis, Tennessee, United States
Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital — Houston, Texas, United States

Full record on ClinicalTrials.gov

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