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



Status:Recruiting
Conditions:Colorectal Cancer, Cancer, Cancer, Brain Cancer, Lymphoma, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:1 - 21
Updated:7/16/2013
Start Date:May 2011

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


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.


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.

DISEASE CHARACTERISTICS:

- 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
days

- 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

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
We found this trial at
21
sites
705 Riley Hospital Drive
Indianapolis, Indiana 46202
317.944.2060
Riley's Children Cancer Center at Riley Hospital for Children The Riley Hospital for Children Cancer...
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1540 East Hospital Drive
Ann Arbor, Michigan 48109
734-936-4000
C.S. Mott Children's Hospital at University of Michigan Medical Center Behind the doors of C.S....
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1405 Clifton Road Northeast
Atlanta, Georgia 30322
(404) 785-6000
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus...
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Atlanta, GA
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1802 6th Avenue South
Birmingham, Alabama 35294
(205) 934-4011
UAB Comprehensive Cancer Center One of the nation’s leading cancer research and treatment centers, the...
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Birmingham, AL
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450 Brookline Ave
Boston, Massachusetts 2215
(617) 632-3000
Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute Founded in 1997, Dana-Farber/Harvard Cancer Center (DF/HCC) was...
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Boston, MA
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3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
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Cincinnati, OH
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Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas From its...
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262 Danny Thomas Pl
Memphis, Tennessee 38105
(901) 495-3300
St. Jude Children's Research Hospital St. Jude is unlike any other pediatric treatment and research...
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Memphis, TN
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701 West 168th Street
New York, New York 10032
(212) 851-4680
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center The Herbert Irving Comprehensive Cancer...
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New York, NY
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1201 W La Veta Ave
Orange, California 92868
(714) 997-3000
Children's Hospital of Orange County For more than 45 years, CHOC Children’s has been steadfastly...
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Orange, CA
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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3181 SW Sam Jackson Park Rd
Portland, Oregon 97239
(503) 494-5058
Knight Cancer Institute at Oregon Health and Science University Cancer takes many forms. Although cancer...
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Portland, OR
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660 S Euclid Ave #8100
Saint Louis, Missouri 63110
(314) 362-5196
Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis The Alvin J. Siteman Cancer Center...
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1600 Divisadero Street
San Francisco, California 94115
888.689.8273
UCSF Helen Diller Family Comprehensive Cancer Center UCSF’s long tradition of excellence in cancer research...
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4800 Sand Point Way Northeast
Seattle, Washington 98105
(206) 987-2000
Children's Hospital and Regional Medical Center - Seattle Seattle Children
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2515 N Clark St
Chicago, Illinois 60614
(312) 227-6060
Children's Memorial Hospital, Chicago Ann & Robert H. Lurie Children
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One Baylor Plaza
Houston, Texas 77030
(713) 798-4951
Baylor University Medical Center - Houston Baylor University Medical Center in Dallas began in 1903...
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9000 W Wisconsin Ave
Milwaukee, Wisconsin 53226
(414) 266-2000
Midwest Children's Cancer Center at Children's Hospital of Wisconsin We are the region's only independent...
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111 Michigan Ave NW
Washington, District of Columbia
(202) 476-5000
Childrens National Medical Center As the nation’s children’s hospital, the mission of Children’s National Medical...
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