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.
Masking: Open Label, Primary Purpose: Treatment
Maximum-tolerated dose and/or recommended phase II dose of imetelstat sodium in children with refractory or recurrent solid tumors or lymphoma
Patrick A. Thompson, MD
Baylor College of Medicine
|Children's Hospital of Philadelphia||Philadelphia, Pennsylvania 19104|
|Children's Hospital of Orange County||Orange, California 92668|
|Children's National Medical Center||Washington, District of Columbia 20010-2970|
|Children's Hospital and Regional Medical Center - Seattle||Seattle, Washington 98105|
|Children's Memorial Hospital - Chicago||Chicago, Illinois 60614|
|St. Jude Children's Research Hospital||Memphis, Tennessee 38105-2794|
|Midwest Children's Cancer Center at Children's Hospital of Wisconsin||Milwaukee, Wisconsin 53226|
|Cincinnati Children's Hospital Medical Center||Cincinnati, Ohio 45229-3039|
|Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas||Dallas, Texas 75390|
|Baylor University Medical Center - Houston||Houston, Texas 77030-2399|
|UCSF Helen Diller Family Comprehensive Cancer Center||San Francisco, California 94115|
|AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus||Atlanta, Georgia 30322|
|Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute||Boston, Massachusetts 02115|
|C.S. Mott Children's Hospital at University of Michigan Medical Center||Ann Arbor, Michigan 48109-0286|
|Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis||St. Louis, Missouri 63110|
|Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center||New York, New York 10032|
|Knight Cancer Institute at Oregon Health and Science University||Portland, Oregon 97239-3098|
|Children's Hospital of Pittsburgh of UPMC||Pittsburgh, Pennsylvania 15213|
|Riley's Children Cancer Center at Riley Hospital for Children||Indianapolis, Indiana 46202-5225|
|UAB Comprehensive Cancer Center||Birmingham, Alabama 35294|
|Warren Grant Magnuson Clinical Center - NCI Clinical Trials Referral Office||Bethesda, Maryland 20892-1182|