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A Phase I Study of Imetelstat, a Telomerase Inhibitor, in Children With Refractory or Recurrent Solid Tumors and Lymphomas

Phase 1
1 Year
21 Years
Open (Enrolling)
Brain and Central Nervous System Tumors, Lymphoma, Lymphoproliferative Disorder, Small Intestine Cancer, Unspecified Childhood Solid Tumor, Protocol Specific

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Trial Information

A Phase I Study of Imetelstat, a Telomerase Inhibitor, in Children With Refractory or Recurrent Solid Tumors and Lymphomas



- To estimate the maximum-tolerated dose (MTD) and/or recommended phase II dose of
imetelstat sodium in children with refractory or recurrent solid tumors or lymphoma.

- To define and describe the toxicities of imetelstat sodium.

- To characterize the pharmacokinetics of imetelstat sodium in children with refractory
or recurrent solid tumors or lymphoma.


- To determine, in a preliminary manner, the antitumor effects of imetelstat sodium in
children with refractory or recurrent solid tumors or lymphoma. (exploratory)

- To provide preliminary assessment of the biological activity of imetelstat sodium in
children with recurrent or refractory malignancies by assessing telomerase activity,
telomere length, hTERT protein, hTERT mRNA, and hTR levels in patient peripheral blood
mononuclear cells (PBMNC) samples pretreatment and on treatment. (Exploratory)

- To assess telomerase activity, hTERT expression, telomere length, hTERT protein, hTERT
mRNA, and hTR levels in patients' pretreatment tumor samples. (Exploratory)

OUTLINE: This is a multicenter, dose-escalation study.

Patients receive imetelstat sodium IV over 2 hours on days 1 and 8. Treatment repeats every
21 days for up to 18 courses in the absence of disease progression or unacceptable toxicity.

Patients undergo blood sample collection at baseline and periodically during study for
pharmacokinetic and correlative studies. Tumor tissue samples from diagnosis and/or
subsequent tumor resections or biopsies may also be collected for correlative studies.

After completion of study therapy, patients are followed up for 30 days.

Inclusion Criteria


- Diagnosis of refractory or recurrent solid tumors, including lymphoma

- No CNS tumors or known CNS metastases (Part A, dose escalation)

- CNS tumors or known CNS metastases allowed (Part B, maximum-tolerated dose or
recommended phase II dose)

- No prior or concurrent CNS hemorrhage on a baseline MRI within the past 14

- All patients must have histologic verification of malignancy at original
diagnosis or relapse except for:

- Intrinsic brain stem tumors

- Optic pathway gliomas

- Pineal tumors and elevations of CSF or serum tumor markers including
alpha-fetoprotein or beta-HCG

- Measurable or evaluable disease

- Disease for which there is no known curative therapy or therapy proven to prolong
survival with an acceptable quality of life

- Patients with known bone marrow metastatic disease will be eligible for study
provided they meet the blood count criteria and they are not known to be refractory
to red cell or platelet transfusions


- Karnofsky performance status (PS) 50-100% (patients > 16 years of age) OR Lansky PS
50-100% (patients ≤ 16 years of age)

- ANC ≥ 1,000/mm³

- Platelet count ≥ 100,000/mm³ (transfusion-independent, defined as not receiving
platelet transfusion within the past 7 days prior to enrollment)

- Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR a serum creatinine based on
age and/or gender as follows:

- 0.6 mg/dL (1 to < 2 years of age)

- 0.8 mg/dL (2 to < 6 years of age)

- 1.0 mg/dL (6 to < 10 years of age)

- 1.2 mg/dL (10 to < 13 years of age)

- 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 to < 16 years of age)

- 1.7 mg/dL (male) or 1.4 mg/dL (female) (≥ 16 years of age)

- Bilirubin (sum of conjugated and unconjugated) ≤ 1.5 times upper limit of normal

- ALT ≤ 110 U/L (ULN for ALT is 45 U/L)

- Serum albumin ≥ 2 g/dL

- aPTT < 1.2 times ULN

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use an effective contraception method

- No uncontrolled infection

- No patients who, in the opinion of the investigator, may not be able to comply with
the safety monitoring requirements of the study


- Recovered from acute toxic effects of all prior anti-cancer chemotherapy,
immunotherapy, or radiotherapy

- At least 3 weeks since prior myelosuppressive chemotherapy (6 weeks for nitrosourea)

- At least 14 days since prior long-acting growth factor (e.g., Neulasta) or ≥ 7 days
since prior short-acting growth factor

- At least 7 days since prior biologic or anti-neoplastic agent

- At least 6 weeks since any type of prior immunotherapy (e.g., tumor vaccines)

- At least 3 half-lives since last dose of a monoclonal antibody

- At least 2 weeks since prior local palliative radiotherapy (small port)

- At least 24 weeks since prior total-body irradiation, craniospinal radiotherapy,
or radiation to ≥ 50% of the pelvis

- At least 6 weeks since prior substantial bone marrow radiation

- At least 12 weeks since prior transplantation or stem cell infusion with no evidence
of active graft vs host disease

- Prior and concurrent stable or decreasing dose of corticosteroids within the past 7
days allowed

- No prior allogeneic transplant

- No other concurrent investigational drug

- No other concurrent anticancer agents including chemotherapy, radiotherapy,
immunotherapy, or biologic therapy

- No concurrent cyclosporine, tacrolimus, or other agents to prevent either
graft-versus-host disease post-bone marrow transplant or organ rejection

Type of Study:


Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Maximum-tolerated dose and/or recommended phase II dose of imetelstat sodium in children with refractory or recurrent solid tumors or lymphoma

Safety Issue:


Principal Investigator

Patrick A. Thompson, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Baylor College of Medicine



Study ID:




Start Date:

May 2011

Completion Date:

Related Keywords:

  • Brain and Central Nervous System Tumors
  • Lymphoma
  • Lymphoproliferative Disorder
  • Small Intestine Cancer
  • Unspecified Childhood Solid Tumor, Protocol Specific
  • unspecified childhood solid tumor, protocol specific
  • recurrent childhood anaplastic large cell lymphoma
  • recurrent childhood grade III lymphomatoid granulomatosis
  • recurrent childhood large cell lymphoma
  • recurrent childhood lymphoblastic lymphoma
  • recurrent childhood small noncleaved cell lymphoma
  • recurrent/refractory childhood Hodgkin lymphoma
  • recurrent childhood brain stem glioma
  • recurrent childhood anaplastic astrocytoma
  • recurrent childhood anaplastic oligoastrocytoma
  • recurrent childhood anaplastic oligodendroglioma
  • recurrent childhood cerebellar astrocytoma
  • recurrent childhood cerebral astrocytoma
  • recurrent childhood diffuse astrocytoma
  • recurrent childhood fibrillary astrocytoma
  • recurrent childhood gemistocytic astrocytoma
  • recurrent childhood giant cell glioblastoma
  • recurrent childhood glioblastoma
  • recurrent childhood gliomatosis cerebri
  • recurrent childhood gliosarcoma
  • recurrent childhood oligoastrocytoma
  • recurrent childhood oligodendroglioma
  • recurrent childhood pilocytic astrocytoma
  • recurrent childhood pilomyxoid astrocytoma
  • recurrent childhood pleomorphic xanthoastrocytoma
  • recurrent childhood protoplasmic astrocytoma
  • recurrent childhood subependymal giant cell astrocytoma
  • recurrent childhood visual pathway and hypothalamic glioma
  • recurrent childhood visual pathway glioma
  • childhood pineal parenchymal tumor
  • recurrent childhood central nervous system embryonal tumor
  • childhood central nervous system choriocarcinoma
  • childhood central nervous system germ cell tumor
  • childhood central nervous system germinoma
  • childhood central nervous system mixed germ cell tumor
  • childhood central nervous system teratoma
  • childhood central nervous system yolk sac tumor
  • recurrent childhood pineoblastoma
  • childhood diffuse large cell lymphoma
  • childhood nasal type extranodal NK/T-cell lymphoma
  • childhood nodular lymphocyte predominant Hodgkin lymphoma
  • angioimmunoblastic T-cell lymphoma
  • childhood Burkitt lymphoma
  • childhood grade III lymphomatoid granulomatosis
  • childhood immunoblastic large cell lymphoma
  • cutaneous B-cell non-Hodgkin lymphoma
  • hepatosplenic T-cell lymphoma
  • intraocular lymphoma
  • noncutaneous extranodal lymphoma
  • peripheral T-cell lymphoma
  • post-transplant lymphoproliferative disorder
  • recurrent cutaneous T-cell non-Hodgkin lymphoma
  • recurrent mycosis fungoides/Sezary syndrome
  • small intestine lymphoma
  • Lymphoma
  • Lymphoproliferative Disorders
  • Nervous System Neoplasms
  • Central Nervous System Neoplasms
  • Duodenal Neoplasms
  • Ileal Neoplasms
  • Jejunal Neoplasms
  • Neoplasms
  • Intestinal Neoplasms



Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Children's Hospital of Orange CountyOrange, California  92668
Children's National Medical CenterWashington, District of Columbia  20010-2970
Children's Hospital and Regional Medical Center - SeattleSeattle, Washington  98105
Children's Memorial Hospital - ChicagoChicago, Illinois  60614
St. Jude Children's Research HospitalMemphis, Tennessee  38105-2794
Midwest Children's Cancer Center at Children's Hospital of WisconsinMilwaukee, Wisconsin  53226
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - DallasDallas, Texas  75390
Baylor University Medical Center - HoustonHouston, Texas  77030-2399
UCSF Helen Diller Family Comprehensive Cancer CenterSan Francisco, California  94115
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston CampusAtlanta, Georgia  30322
Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer InstituteBoston, Massachusetts  02115
C.S. Mott Children's Hospital at University of Michigan Medical CenterAnn Arbor, Michigan  48109-0286
Siteman Cancer Center at Barnes-Jewish Hospital - Saint LouisSt. Louis, Missouri  63110
Herbert Irving Comprehensive Cancer Center at Columbia University Medical CenterNew York, New York  10032
Knight Cancer Institute at Oregon Health and Science UniversityPortland, Oregon  97239-3098
Children's Hospital of Pittsburgh of UPMCPittsburgh, Pennsylvania  15213
Riley's Children Cancer Center at Riley Hospital for ChildrenIndianapolis, Indiana  46202-5225
UAB Comprehensive Cancer CenterBirmingham, Alabama  35294
Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral OfficeBethesda, Maryland  20892-1182