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Imetelstat Sodium in Treating Younger Patients With Recurrent or Refractory Brain Tumors

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT01836549
Recruitment Status : Terminated (Due to several intracranial hemorrhages and recommendation by the PBTC DSMB.)
First Posted : April 22, 2013
Results First Posted : July 20, 2018
Last Update Posted : July 20, 2018
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Pediatric Brain Tumor Consortium

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Anaplastic Astrocytoma
Anaplastic Ependymoma
Astrocytoma, Grade II
Ependymoma
Giant Cell Glioblastoma
Glioblastoma
Gliosarcoma
Oligodendroglioma
Brainstem Tumors
Intervention Drug: imetelstat sodium
Enrollment 43
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Molecular Biology Stratum-A Molecular Biology Stratum-B Molecular Biology Stratum-C Stratum-A Stratum-B Stratum-C Stratum-D
Hide Arm/Group Description This is the Molecular Biology arm for Medulloblastoma/PNET Patients This is the Molecular Biology arm for High-Grade Glioma Patients This is the Molecular Biology arm for Ependymoma Patients This is the Phase-II stratum for Recurrent or Refractory Medulloblastoma/PNET Patients This is the Phase-II stratum for Recurrent or refractory high-grade glioma patients This is the Phase-II stratum for Recurrent or refractory ependymoma patients This is the Phase-II stratum for Recurrent or refractory diffuse intrinsic pontine gliomas (DIPG) patients
Period Title: Overall Study
Started 1 1 1 9 18 4 9
Completed 1 1 0 8 18 4 8
Not Completed 0 0 1 1 0 0 1
Reason Not Completed
Did not receive study drug             0             0             1             0             0             0             0
Patient declared ineligible             0             0             0             1             0             0             0
Patient inevaluable to start therapy             0             0             0             0             0             0             1
Arm/Group Title Molecular Biology Stratum-A Molecular Biology Stratum-B Stratum-A Stratum-B Stratum-C Stratum-D Total
Hide Arm/Group Description This is the Molecular Biology arm for Medulloblastoma/PNET Patients This is the Molecular Biology arm for High-Grade Glioma Patients This is the Phase-II stratum for Recurrent or Refractory Medulloblastoma/PNET Patients This is the Phase-II stratum for Recurrent or refractory high-grade glioma patients This is the Phase-II stratum for Recurrent or refractory ependymoma patients This is the Phase-II stratum for Recurrent or refractory diffuse intrinsic pontine gliomas (DIPG) patients Total of all reporting groups
Overall Number of Baseline Participants 1 1 8 18 4 8 40
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 1 participants 1 participants 8 participants 18 participants 4 participants 8 participants 40 participants
10.41 [1]   (NA) 11.33 [1]   (NA) 7.94  (4.31) 11.66  (5.24) 11.51  (3.84) 10.45  (2.14) 10.62  (4.40)
[1]
Only one patient
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 1 participants 1 participants 8 participants 18 participants 4 participants 8 participants 40 participants
Female
0
   0.0%
0
   0.0%
2
  25.0%
7
  38.9%
2
  50.0%
4
  50.0%
15
  37.5%
Male
1
 100.0%
1
 100.0%
6
  75.0%
11
  61.1%
2
  50.0%
4
  50.0%
25
  62.5%
1.Primary Outcome
Title Numver of Patients With Telomerase-positive Archival Tumors Who Demonstrate at Least 50% Reduction
Hide Description This outcome measure is for the Molecular biology study only. The assessment was done to identify cases with at least 50% reduction in telomerase activity.
Time Frame Up to 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
This outcome measure is only for the molecular biology study. Telomerase inhibition was assessed and the inhibition level was compared with the baseline level to identify increased inhibition.
Arm/Group Title Molecular Biology Study
Hide Arm/Group Description:

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.

imetelstat sodium: Given IV

Overall Number of Participants Analyzed 2
Measure Type: Count of Participants
Unit of Measure: Participants
1
  50.0%
2.Primary Outcome
Title Phase II: Stratum-specific Objective Response (CR+PR) Rate
Hide Description

Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), >=50% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR sustained for at least 6 weeks.

For each stratum separately exact confidence interval estimates will be provided for the true, unknown rates of objective response. Estimated by cumulative incidence functions.

Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
This is the Phase-II study cohort, excluding the molecular study patients.
Arm/Group Title Stratum-A Stratum-B Stratum-C Stratum-D
Hide Arm/Group Description:
This is the Phase-II stratum for Recurrent or Refractory Medulloblastoma/PNET Patients
This is the Phase-II stratum for Recurrent or refractory high-grade glioma patients
This is the Phase-II stratum for Recurrent or refractory ependymoma patients
This is the Phase-II stratum for Recurrent or refractory diffuse intrinsic pontine glioma patients
Overall Number of Participants Analyzed 8 18 4 8
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
3.Secondary Outcome
Title Number of Participants With Telomerase Inhibition
Hide Description This outcome measure is applicable only for the Molecular biology study arm. Telomerase inhibition was assessed in PBMCs and summarised as 'yes-inhibited' vs. 'no inhibition'
Time Frame Up to 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
Samples from six patients were consequently considered evaluable and were analyzed.
Arm/Group Title Molecular Biology Study
Hide Arm/Group Description:

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.

imetelstat sodium: Given IV

Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
5
  83.3%
4.Secondary Outcome
Title Stratum-specific Progression-free Survival (PFS) (Phase II)
Hide Description 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.
Time Frame Up to 2 years, 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
Hide Outcome Measure Data
Hide Analysis Population Description
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Arm/Group Title Stratum-A Stratum-B Stratum-C Stratum-D
Hide Arm/Group Description:
This is the Phase-II stratum for Recurrent or Refractory Medulloblastoma/PNET Patients
This is the Phase-II stratum for Recurrent or refractory high-grade glioma patients
This is the Phase-II stratum for Recurrent or refractory ependymoma patients
This is the Phase-II stratum for Recurrent or refractory diffuse intrinsic pontine gliomas (DIPG) patients
Overall Number of Participants Analyzed 8 18 4 8
Mean (Standard Error)
Unit of Measure: days
30  (3.06) 54.2  (9.97) 50.3  (16.25) 52.6  (14.50)
5.Secondary Outcome
Title Number of Patients With Telomerase Expression Data by Detection of hTERT mRNA and TERC RNA Levels by qRT-PCR and Telomerase Activity by TRAP in Archival Tumor Tissue and to Explore Association of Telomerase Positivity With Objective Response and PFS
Hide Description This secondary objective is for Stratum-B and C only, which enroll HGG and ependymoma patients. We will describe the evidence of telomerase expression by detection of hTERT mRNA and TERC RNA levels by qRT-PCR and telomerase activity by TRAP in archival tumor tissue; Association of telomerase positivity with objective response and PFS will not be able to be conducted as the study was terminated early and there was no objective response.
Time Frame Up to 30 days. Due to small number of patients evaluable for this objective, we can only provide number of patients with the targetted markers as no analysis with PFS is possible.
Hide Outcome Measure Data
Hide Analysis Population Description
This objective is for STRATUM-C patients only, having only 4 patients with TRAP, TERT and TERC levels measured. Due to small sample and having no objective response, association studies of these markers with Objective Reponse and PFS were not possible. Therefore, we only report the the percentage of cases with at least weak TRAP activity.
Arm/Group Title Stratum-C
Hide Arm/Group Description:
This objective is for HGG and ependymoma patients only.
Overall Number of Participants Analyzed 4
Measure Type: Count of Participants
Unit of Measure: Participants
TRAP
3
  75.0%
TERT
3
  75.0%
TERC
3
  75.0%
6.Secondary Outcome
Title Quantitative MRI Parameters of Tumors Prior to and After Treatment With Imetelstat (Molecular Biology and Phase II Studies)
Hide Description This outcome measure was for both Molecular biology and Phase II studies. We will not be able to present the results of this objective as the study was terminated early.
Time Frame Up to 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
This study has been terminated early and the study team decided not to pursue this aim as the results would be inconclusive due to small sample size. Neither the neuroimaging, nor the biology data are available for these patients.
Arm/Group Title Treatment (Imetelstat Sodium)
Hide Arm/Group Description:

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.

imetelstat sodium: Given IV

Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame Up to 2 years, which is the total possible therapy duration.
Adverse Event Reporting Description The AEs we report were experienced while the participants were on study.
 
Arm/Group Title Molecular Study-A Molecular Study-B Stratum-A Stratum-B Stratum-C Stratum-D
Hide Arm/Group Description Molecular Study arm for Recurrent or Refractory Medulloblastoma/PNET Patients Molecular Study arm for Recurrent or refractory high-grade glioma patients This is the Phase-II stratum for Recurrent or Refractory Medulloblastoma/PNET Patients This is the Phase-II stratum for Recurrent or refractory high-grade glioma patients This is the Phase-II stratum for Recurrent or refractory ependymoma patients This is the Phase-II stratum for Recurrent or refractory diffuse intrinsic pontine gliomas (DIPG) patients
All-Cause Mortality
Molecular Study-A Molecular Study-B Stratum-A Stratum-B Stratum-C Stratum-D
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Molecular Study-A Molecular Study-B Stratum-A Stratum-B Stratum-C Stratum-D
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/1 (100.00%)      0/1 (0.00%)      3/8 (37.50%)      4/18 (22.22%)      2/4 (50.00%)      3/8 (37.50%)    
Gastrointestinal disorders             
Dysphagia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
General disorders             
Fatigue  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Death, NOS  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 1/4 (25.00%)  1 0/8 (0.00%)  0
Investigations             
Alanine aminotransferase increased  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Aspartate aminotransferase increased  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Platelet count decreased  1  1/1 (100.00%)  1 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Metabolism and nutrition disorders             
Hyponatremia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Musculoskeletal and connective tissue disorders             
Muscle weakness upper limb  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify[Death due to pr  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 2/8 (25.00%)  2
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify[Progressive dis  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 1/8 (12.50%)  1
Intracranial hemorrhage  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 3/18 (16.67%)  3 0/4 (0.00%)  0 0/8 (0.00%)  0
Nervous system disorders             
Depressed level of consciousness  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Dysarthria  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Hydrocephalus  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypoglossal nerve disorder  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Nervous system disorders - Other, specify[Cervical hemorrhage]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Seizure  1  1/1 (100.00%)  1 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Syncope  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Psychiatric disorders             
Confusion  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders             
Apnea  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Aspiration  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Dyspnea  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Respiratory failure  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders - Other, specify[increased secretions requiring freq  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (14.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Molecular Study-A Molecular Study-B Stratum-A Stratum-B Stratum-C Stratum-D
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/1 (100.00%)      1/1 (100.00%)      8/8 (100.00%)      16/18 (88.89%)      4/4 (100.00%)      8/8 (100.00%)    
Blood and lymphatic system disorders             
Anemia  1  1/1 (100.00%)  1 0/1 (0.00%)  0 4/8 (50.00%)  4 7/18 (38.89%)  7 2/4 (50.00%)  2 0/8 (0.00%)  0
Cardiac disorders             
Sinus tachycardia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Eye disorders             
Eye disorders - Other, specify[Diplopia]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Scleral disorder  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Gastrointestinal disorders             
Abdominal pain  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Constipation  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 1/4 (25.00%)  1 0/8 (0.00%)  0
Dental caries  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Diarrhea  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Fecal incontinence  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Nausea  1  0/1 (0.00%)  0 0/1 (0.00%)  0 3/8 (37.50%)  3 2/18 (11.11%)  2 1/4 (25.00%)  1 2/8 (25.00%)  2
Vomiting  1  0/1 (0.00%)  0 0/1 (0.00%)  0 2/8 (25.00%)  2 5/18 (27.78%)  5 1/4 (25.00%)  1 2/8 (25.00%)  2
General disorders             
Fatigue  1  0/1 (0.00%)  0 1/1 (100.00%)  1 4/8 (50.00%)  4 4/18 (22.22%)  4 1/4 (25.00%)  1 3/8 (37.50%)  3
Fever  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 1/4 (25.00%)  1 1/8 (12.50%)  1
Infusion related reaction  1  0/1 (0.00%)  0 0/1 (0.00%)  0 3/8 (37.50%)  3 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Localized edema  1  0/1 (0.00%)  0 1/1 (100.00%)  1 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Non-cardiac chest pain  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Pain  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Immune system disorders             
Allergic reaction  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Infections and infestations             
Papulopustular rash  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Upper respiratory infection  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Investigations             
Activated partial thromboplastin time prolonged  1  1/1 (100.00%)  1 0/1 (0.00%)  0 1/8 (12.50%)  1 3/18 (16.67%)  3 0/4 (0.00%)  0 1/8 (12.50%)  1
Alanine aminotransferase increased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 4/8 (50.00%)  4 8/18 (44.44%)  8 0/4 (0.00%)  0 6/8 (75.00%)  6
Alkaline phosphatase increased  1  0/1 (0.00%)  0 1/1 (100.00%)  1 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Aspartate aminotransferase increased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 4/8 (50.00%)  4 7/18 (38.89%)  7 0/4 (0.00%)  0 3/8 (37.50%)  3
Blood bilirubin increased  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hemoglobin increased  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Investigations - Other, specify[increased PT]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Lymphocyte count decreased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 5/8 (62.50%)  5 8/18 (44.44%)  8 0/4 (0.00%)  0 6/8 (75.00%)  6
Lymphocyte count increased  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Neutrophil count decreased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 4/8 (50.00%)  4 9/18 (50.00%)  9 2/4 (50.00%)  2 1/8 (12.50%)  1
Platelet count decreased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 8/8 (100.00%)  8 11/18 (61.11%)  11 1/4 (25.00%)  1 5/8 (62.50%)  5
Weight loss  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
White blood cell decreased  1  1/1 (100.00%)  1 1/1 (100.00%)  1 3/8 (37.50%)  3 8/18 (44.44%)  8 1/4 (25.00%)  1 1/8 (12.50%)  1
Metabolism and nutrition disorders             
Anorexia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 2/8 (25.00%)  2 3/18 (16.67%)  3 0/4 (0.00%)  0 0/8 (0.00%)  0
Dehydration  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Hyperkalemia  1  0/1 (0.00%)  0 1/1 (100.00%)  1 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypermagnesemia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 2/18 (11.11%)  2 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypernatremia  1  0/1 (0.00%)  0 1/1 (100.00%)  1 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypoalbuminemia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 2/18 (11.11%)  2 0/4 (0.00%)  0 1/8 (12.50%)  1
Hypocalcemia  1  0/1 (0.00%)  0 1/1 (100.00%)  1 3/8 (37.50%)  3 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypoglycemia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypokalemia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 3/8 (37.50%)  3
Hyponatremia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 2/8 (25.00%)  2
Hypophosphatemia  1  0/1 (0.00%)  0 1/1 (100.00%)  1 2/8 (25.00%)  2 1/18 (5.56%)  1 0/4 (0.00%)  0 2/8 (25.00%)  2
Musculoskeletal and connective tissue disorders             
Arthralgia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Muscle weakness left-sided  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 2/18 (11.11%)  2 0/4 (0.00%)  0 0/8 (0.00%)  0
Musculoskeletal and connective tissue disorder - Other, specify[Foot pain]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Myalgia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)             
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 1/4 (25.00%)  1 1/8 (12.50%)  1
Nervous system disorders             
Abducens nerve disorder  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Ataxia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 2/18 (11.11%)  2 0/4 (0.00%)  0 2/8 (25.00%)  2
Dysarthria  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 1/18 (5.56%)  1 0/4 (0.00%)  0 2/8 (25.00%)  2
Dysesthesia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Dysphasia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 2/8 (25.00%)  2
Facial nerve disorder  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 1/18 (5.56%)  1 1/4 (25.00%)  1 0/8 (0.00%)  0
Headache  1  0/1 (0.00%)  0 1/1 (100.00%)  1 3/8 (37.50%)  3 4/18 (22.22%)  4 0/4 (0.00%)  0 2/8 (25.00%)  2
Hypoglossal nerve disorder  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 2/18 (11.11%)  2 0/4 (0.00%)  0 0/8 (0.00%)  0
Intracranial hemorrhage  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Lethargy  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Nervous system disorders - Other, specify[Inability to tolerate own secretions]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Paresthesia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Seizure  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Tremor  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Psychiatric disorders             
Agitation  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Confusion  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Depression  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Insomnia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Suicidal ideation  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Renal and urinary disorders             
Renal and urinary disorders - Other, specify[Urinary Hesitancy]  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Urinary frequency  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Urinary incontinence  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Urinary retention  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Respiratory, thoracic and mediastinal disorders             
Bronchospasm  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Dyspnea  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Epistaxis  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Skin and subcutaneous tissue disorders             
Alopecia  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Pruritus  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Vascular disorders             
Flushing  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 1/18 (5.56%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Hot flashes  1  0/1 (0.00%)  0 0/1 (0.00%)  0 0/8 (0.00%)  0 0/18 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Hypertension  1  0/1 (0.00%)  0 0/1 (0.00%)  0 1/8 (12.50%)  1 0/18 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (14.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Dr. Mehmet Kocak
Organization: University of Tennessee Health Science Center
Phone: 9014482937 ext 901
Responsible Party: Pediatric Brain Tumor Consortium
ClinicalTrials.gov Identifier: NCT01836549     History of Changes
Other Study ID Numbers: PBTC-036
NCI-2013-00482 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
U01CA081457 ( U.S. NIH Grant/Contract )
First Submitted: April 17, 2013
First Posted: April 22, 2013
Results First Submitted: December 13, 2016
Results First Posted: July 20, 2018
Last Update Posted: July 20, 2018