NCT01273090

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

A Phase 1 Study of Imetelstat, a Telomerase Inhibitor, in Children With Refractory or Recurrent Solid Tumors and Lymphomas

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Children's Oncology Group · Network
• Sex: All
• Age: 1 Year – 21 Years
• Healthy volunteers: Not accepted

Summary

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

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

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

Interventions

Type	Name	Description
DRUG	imetelstat sodium
OTHER	laboratory biomarker analysis
OTHER	pharmacological study

Timeline

• Start date: 2011-05-01
• Primary completion: 2013-09-01
• Completion: 2013-10-01
• First posted: 2011-01-10
• Last updated: 2014-01-30

Locations

23 sites across 2 countries: United States, Canada