Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors
|ClinicalTrials.gov Identifier: NCT01836549|
Recruitment Status : Completed
First Posted : April 22, 2013
Last Update Posted : April 12, 2016
|Condition or disease||Intervention/treatment||Phase|
|Anaplastic Astrocytoma Anaplastic Ependymoma Astrocytoma, Grade II Ependymoma Giant Cell Glioblastoma Glioblastoma Gliosarcoma Oligodendroglioma Brainstem Tumors||Drug: imetelstat sodium||Phase 2|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||43 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Molecular Biology and Phase II Study of Imetelstat (GRN163L) in Children With Recurrent High-Grade Glioma, Ependymoma and Diffuse Intrinsic Pontine Glioma|
|Study Start Date :||March 2013|
|Primary Completion Date :||April 2016|
|Study Completion Date :||April 2016|
Experimental: Treatment (imetelstat sodium)
Molecular Biology Phase: Patients will receive one infusion of imetelstat prior to surgery. Surgery will take place 12-24 hours after the infusion of imetelstat. Patients will continue to receive therapy on the same schedule as the Phase II patients starting 14-21 days after surgery.
Phase II: Patients receive imetelstat sodium IV over 2 hours on days 1 and 8. Treatment repeats every 21 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Drug: imetelstat sodium
- Molecular biology study: Percentage of subjects with telomerase-positive archival tumors who demonstrate at least 50% reduction in telomerase activity [ Time Frame: Up to 30 days ]Summarized and described via summary statistics and plots.
- Phase II: Stratum-specific objective response (CR+PR) rate [ Time Frame: 6 months ]For each stratum separately exact confidence interval estimates will be provided for the true, unknown rates of objective response. Estimated by cumulative incidence functions.
- Quantitative assessments of hTERT mRNA and TERC RNA levels (Molecular biology study) [ Time Frame: Up to 30 days ]Summarized and described via summary statistics and plots. Descriptive statistics, plots and, if adequate data are available to make such models viable, mixed effects models and changes in telomerase activity in peripheral blood mononuclear cells (PBMNCs) longitudinally will be explored.
- Stratum-specific progression-free survival (PFS) (Phase II) [ Time Frame: From the date of initial treatment to the earliest date of disease progression, second malignancy or death for subjects who fail; and to the date of last contact for subjects who remain at risk for failure, assessed up to 3 years ]Kaplan-Meier estimates of distributions of survival and PFS for all eligible subjects who received at least one dose of imetelstat will be provided separately. Similarly, Cox regression models may be used to look for associations between PK parameters and PFS separately in each stratum in the Phase II study.
- Quantitative assessment of telomerase activity by TRAP and telomere length by Southern blot (Molecular biology and Phase II studies) [ Time Frame: Up to 30 days ]95% confidence intervals will be estimated. Summarized and described via summary statistics and plots.
- ALT use by TRF analysis/Southern blot, telomere-specific FISH and localization of ATRX/DAXX (Molecular biology and Phase II studies) [ Time Frame: Up to 30 days ]Summarized and described via summary statistics and plots.
- Quantitative MRI parameters of tumors prior to and after treatment with imetelstat (Molecular biology and Phase II studies) [ Time Frame: Up to 30 days ]Summarized and described via summary statistics and plots.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01836549
|United States, California|
|Childrens Hospital Los Angeles|
|Los Angeles, California, United States, 90027-0700|
|Lucile Packard Children's Hospital at Stanford University Medical Center|
|Palo Alto, California, United States, 94304|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010-2970|
|United States, Illinois|
|Lurie Children's Hospital- Chicago|
|Chicago, Illinois, United States, 60614|
|United States, Maryland|
|NCI - Pediatric Oncology Branch|
|Bethesda, Maryland, United States, 20892|
|United States, New York|
|Memorial Sloan-Kettering Cancer Center|
|New York, New York, United States, 10065|
|United States, North Carolina|
|Duke Comprehensive Cancer Center|
|Durham, North Carolina, United States, 27710|
|United States, Ohio|
|Cincinnati Children's Hospital Medical Center|
|Cincinnati, Ohio, United States, 45229-3039|
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh|
|Pittsburgh, Pennsylvania, United States, 15213|
|United States, Tennessee|
|Saint Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38105-2794|
|United States, Texas|
|Texas Children's Cancer Center and Hematology Service at Texas Children's Hospital|
|Houston, Texas, United States, 77030-2399|
|Principal Investigator:||Maryam Fouladi||Pediatric Brain Tumor Consortium|