Imetelstat Sodium in Treating Young Patients With Refractory or Recurrent Solid Tumors or Lymphoma

NCT ID: NCT01273090

Last Updated: 2014-01-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

34 participants

Study Classification

INTERVENTIONAL

Study Start Date

2011-05-31

Study Completion Date

2013-10-31

Brief Summary

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RATIONALE: Imetelstat sodium may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

PURPOSE: This phase I clinical trial is studying the side effects and best dose of imetelstat sodium in treating young patients with refractory or recurrent solid tumors or lymphoma.

Detailed Description

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OBJECTIVES:

Primary

* 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.

Secondary

* 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.

Conditions

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Brain and Central Nervous System Tumors Lymphoma Lymphoproliferative Disorder Small Intestine Cancer Unspecified Childhood Solid Tumor, Protocol Specific

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Treatment

Group Type EXPERIMENTAL

imetelstat sodium

Intervention Type DRUG

laboratory biomarker analysis

Intervention Type OTHER

pharmacological study

Intervention Type OTHER

Interventions

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imetelstat sodium

Intervention Type DRUG

laboratory biomarker analysis

Intervention Type OTHER

pharmacological study

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

PATIENT CHARACTERISTICS:

* 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 (ULN)
* 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

PRIOR CONCURRENT THERAPY:

* 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 post-transplant
Minimum Eligible Age

1 Year

Maximum Eligible Age

21 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Cancer Institute (NCI)

NIH

Sponsor Role collaborator

Children's Oncology Group

NETWORK

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Patrick A. Thompson, MD

Role: PRINCIPAL_INVESTIGATOR

Baylor College of Medicine

Locations

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UAB Comprehensive Cancer Center

Birmingham, Alabama, United States

Site Status

Children's Hospital of Orange County

Orange, California, United States

Site Status

UCSF Helen Diller Family Comprehensive Cancer Center

San Francisco, California, United States

Site Status

Children's National Medical Center

Washington D.C., District of Columbia, United States

Site Status

AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus

Atlanta, Georgia, United States

Site Status

Children's Memorial Hospital - Chicago

Chicago, Illinois, United States

Site Status

Riley's Children Cancer Center at Riley Hospital for Children

Indianapolis, Indiana, United States

Site Status

Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office

Bethesda, Maryland, United States

Site Status

Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute

Boston, Massachusetts, United States

Site Status

C.S. Mott Children's Hospital at University of Michigan Medical Center

Ann Arbor, Michigan, United States

Site Status

Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis

St Louis, Missouri, United States

Site Status

Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center

New York, New York, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Knight Cancer Institute at Oregon Health and Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas

Dallas, Texas, United States

Site Status

Baylor University Medical Center - Houston

Houston, Texas, United States

Site Status

Children's Hospital and Regional Medical Center - Seattle

Seattle, Washington, United States

Site Status

Midwest Children's Cancer Center at Children's Hospital of Wisconsin

Milwaukee, Wisconsin, United States

Site Status

Hospital for Sick Children

Toronto, Ontario, Canada

Site Status

Hopital Sainte Justine

Montreal, Quebec, Canada

Site Status

Countries

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United States Canada

Other Identifiers

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COG-ADVL1112

Identifier Type: -

Identifier Source: secondary_id

ADVL1112

Identifier Type: -

Identifier Source: org_study_id

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