Clinical Trial Details
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NCT01836549 : Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors
PhasePhase 2
AgesMin: 12 Months Max: 21 Years


- Tumor: Subjects must have a histologically confirmed diagnosis 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.


- Tumor: Subjects must have recurrent or refractory disease with a histological
diagnosis 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 diagnosis 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.


- Subjects with neurological deficits should have deficits that are stable for a
minimum of one (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

- Age 2 to < 6 years: maximum serum creatinine (mg/mL) 0.8 for males and 0.8 for

- 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

- Age 13 to < 16 years: maximum serum creatinine (mg/mL) 1.5 for males and 1.4 for

- Age >= 16 years: maximum serum creatinine (mg/mL) 1.7 for males and 1.4 for females

- The threshold creatinine values were derived from the Schwartz formula for estimating
GFR (Schwartz et al. J. Peds, 106:522, 1985) utilizing child length and stature data
published by the Centers for Disease and Control (CDC)

- Subjects on systemic anticoagulants are excluded from this study as the drug can
cause minor, transient changes in aPTT

- Female subjects of childbearing potential must not be pregnant or
breast-feeding; female subjects of childbearing potential must have a negative
serum or urine pregnancy test; (pregnancy test must be repeated within 48 hours
prior to the start of therapy)

- Subjects of childbearing or child fathering potential must be willing to use a
medically acceptable form of birth control, which includes abstinence, while
being treated on this study

- Subjects must have recovered from the acute toxicities of all prior therapy
before entering this study; for those acute baseline adverse events attributable
to prior therapy, recovery is defined as a toxicity grade =< 2, using Common
Terminology Criteria for Adverse Events (CTCAE) v.4.0, unless otherwise
specified in the Inclusion and Exclusion Criteria

- Subjects must have received their last dose of known myelosuppressive anticancer
chemotherapy at least three (3) weeks prior to study registration or at least
six (6) weeks if nitrosourea

- Subjects must have received their last dose of investigational or biologic agent
>= 7 days prior to study registration; in the event that a subject has received
an investigational or biologic agent and has experienced >= grade 2
myelosuppression, then at least three (3) weeks must have elapsed prior to
registration; if the investigational or biologic agent has a prolonged half-life
(>= 7 days) then at least three (3) weeks must have elapsed prior to

- Subjects must have completed at least 3 half-life periods from the last dose of
monoclonal antibody prior to registration; Note: A list of half-lives of
commonly used monoclonal antibodies is available on the Pediatric Brain Tumor
Consortium (PBTC) website under Generic Forms and Templates

- Subjects must have received their last dose of radiation (XRT):

- 2 weeks prior to study registration for local palliative XRT (small volume)

- 3 months prior to study registration for craniospinal XRT

- 6 weeks (wks) prior to study registration for other substantial bone marrow

- Subject must be >= 3 months since autologous bone marrow/stem cell
transplantation prior to registration

- Subjects who are receiving a corticosteroid, such as dexamethasone, must be on a
stable or decreasing dosage for at least 1 week prior to registration

- At least 7 days since the completion of therapy with a hematopoietic growth
agent (filgrastim, sargramostim, and erythropoietin) and 14 days for long-acting

- Ability to understand and the willingness to sign a written informed consent


- 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
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