Trial Outcomes & Findings for Modafinil in Treating Children With Memory and Attention Problems Caused by Cancer Treatment for a Brain Tumor (NCT NCT01381718)
NCT ID: NCT01381718
Last Updated: 2021-08-13
Results Overview
CogState Battery. CogState is a semi-automated, computerized cognitive testing system that was developed as a rapid and accurate test of cognitive function specifically for repeated assessment that is sensitive to the effects of medication in children over the age of 5 years and in adults from different language, cultural and socio-economic backgrounds. The CogState tasks to assess processing speed, visual attention, working memory and executive function were used. The CogState battery was administered in the following order: Detection Task, Identification Task, One Card Learning Task, One Back Task, and lastly, the Modified Groton Maze Learning Task. It was administered at baseline and 6 weeks. A positive change from baseline is an improvement. There is not a minimum or maximum since the value is reported as a z-score, but with the mean = 0 and the SD = 1, the range should be between -3 and 3.
COMPLETED
PHASE2
112 participants
Baseline and 6 weeks
2021-08-13
Participant Flow
Participant milestones
| Measure |
Arm I - Modafinil
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Overall Study
STARTED
|
56
|
56
|
|
Overall Study
COMPLETED
|
51
|
43
|
|
Overall Study
NOT COMPLETED
|
5
|
13
|
Reasons for withdrawal
| Measure |
Arm I - Modafinil
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
2
|
5
|
|
Overall Study
Lost to Follow-up
|
3
|
5
|
|
Overall Study
Toxicity, side effects or complications related to study
|
0
|
3
|
Baseline Characteristics
Modafinil in Treating Children With Memory and Attention Problems Caused by Cancer Treatment for a Brain Tumor
Baseline characteristics by cohort
| Measure |
Arm I - Modafanil
n=56 Participants
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=56 Participants
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
Total
n=112 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
12.12 Years
STANDARD_DEVIATION 2.92 • n=5 Participants
|
12.7 Years
STANDARD_DEVIATION 3.12 • n=7 Participants
|
12.41 Years
STANDARD_DEVIATION 3.02 • n=5 Participants
|
|
Sex: Female, Male
Female
|
27 Participants
n=5 Participants
|
31 Participants
n=7 Participants
|
58 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
29 Participants
n=5 Participants
|
25 Participants
n=7 Participants
|
54 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
8 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
45 Participants
n=5 Participants
|
46 Participants
n=7 Participants
|
91 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
6 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
13 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
47 Participants
n=5 Participants
|
43 Participants
n=7 Participants
|
90 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
BMI
|
22.66 kg/m^2
STANDARD_DEVIATION 7.53 • n=5 Participants
|
21.5 kg/m^2
STANDARD_DEVIATION 6.55 • n=7 Participants
|
22.08 kg/m^2
STANDARD_DEVIATION 7.05 • n=5 Participants
|
|
Weight in kg
|
49.68 Kg
STANDARD_DEVIATION 25.72 • n=5 Participants
|
47.72 Kg
STANDARD_DEVIATION 20.96 • n=7 Participants
|
48.7 Kg
STANDARD_DEVIATION 23.38 • n=5 Participants
|
|
Height in cm
|
143.93 cm
STANDARD_DEVIATION 17.34 • n=5 Participants
|
149.74 cm
STANDARD_DEVIATION 39.03 • n=7 Participants
|
146.84 cm
STANDARD_DEVIATION 30.21 • n=5 Participants
|
|
Primary Cancer Diagnosis
Medulloblastoma PNET or ATRT
|
22 Participants
n=5 Participants
|
29 Participants
n=7 Participants
|
51 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Pilocytic Astrocytoma (Grade 1)
|
10 Participants
n=5 Participants
|
10 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Ependymoma
|
7 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Germinoma or non-germinomatous germ
|
4 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Astrocytoma (Grade 2)
|
3 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
6 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Other
|
4 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
8 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Craniopharygioma
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Optic Pathway Tumor (optic nerve, chiasmatic optic tract)
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Choroid plexus tumor (all grades)
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Oligodendroglioma
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Pinealoma (all grades)
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Primary Cancer Diagnosis
Missing
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline and 6 weeksPopulation: Decreased number of participants analyzed is because not all participants had a CogState score at 6 weeks
CogState Battery. CogState is a semi-automated, computerized cognitive testing system that was developed as a rapid and accurate test of cognitive function specifically for repeated assessment that is sensitive to the effects of medication in children over the age of 5 years and in adults from different language, cultural and socio-economic backgrounds. The CogState tasks to assess processing speed, visual attention, working memory and executive function were used. The CogState battery was administered in the following order: Detection Task, Identification Task, One Card Learning Task, One Back Task, and lastly, the Modified Groton Maze Learning Task. It was administered at baseline and 6 weeks. A positive change from baseline is an improvement. There is not a minimum or maximum since the value is reported as a z-score, but with the mean = 0 and the SD = 1, the range should be between -3 and 3.
Outcome measures
| Measure |
Arm I - Modafinil
n=50 Participants
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=42 Participants
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Change in Age-Adjusted Scores at Week Six From Baseline in the Attention Task of the CogState Battery
|
-0.16 Z Score
Standard Deviation 0.71
|
0.14 Z Score
Standard Deviation 1.06
|
SECONDARY outcome
Timeframe: 30 days post interventionAEs gathered using SAFTEE (Systematic Assessment for Treatment Emergent Effects) and participant report on daily log.
Outcome measures
| Measure |
Arm I - Modafinil
n=56 Participants
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=56 Participants
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Number of Reported Adverse Events (AEs)
|
76 Adverse Events
|
26 Adverse Events
|
SECONDARY outcome
Timeframe: Baseline and 6 weeksPopulation: Not all participants had a BRIEF score at 6 weeks
Behavior Rating Inventory of Executive Function (BRIEF). The BRIEF is a behavior rating scale designed to assess executive functions in the home and school environments. The measure was selected to provide parent-reported outcomes of problems related to attention, memory and executive function that occur in everyday life. The instrument was utilized to measure the change in working memory, according to the parent's perspective only, from baseline to 6 weeks. Scores are linear transformations of raw scores into T scores (mean = 50, SD = 10); higher scores indicate greater difficulties. The baseline score was subtracted from the 6 week score. Positive change from baseline is an improvement. The total scale ranges from 0 to 172. A T score \>60 on the BRIEF working memory subscale indicates cognitive impairment.
Outcome measures
| Measure |
Arm I - Modafinil
n=51 Participants
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=43 Participants
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Change in Working Memory Score at 6 Weeks From Baseline as Assessed on BRIEF
|
-4.1 T Score
Standard Deviation 7.4
|
-4.5 T Score
Standard Deviation 9.2
|
SECONDARY outcome
Timeframe: Baseline and 6 weeksPopulation: Not all participants had a completed PedsQL at Week 6
PedsQL is Pediatric Quality of Life Inventory Multi-dimensional Fatigue Scale. This scale is designed as a generic symptom-specific instrument to measure fatigue. Higher scores indicate fewer symptoms of fatigue. It was administered at baseline and 6 weeks. The score is the sum of the answers. The baseline score was subtracted from the 6 week score. A positive change indicates worsening. The scale total score range is from 0-72 for both the parent and patient reported instruments.
Outcome measures
| Measure |
Arm I - Modafinil
n=51 Participants
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=43 Participants
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Change in PedsQL Score at 6 Weeks From Baseline
Parent Rating
|
11.3 Scores on a scale
Standard Deviation 17.6
|
11.4 Scores on a scale
Standard Deviation 15.9
|
|
Change in PedsQL Score at 6 Weeks From Baseline
Patient Rating
|
9.3 Scores on a scale
Standard Deviation 16.5
|
6.9 Scores on a scale
Standard Deviation 16.6
|
Adverse Events
Arm I - Modafanil
Arm II - Placebo
Serious adverse events
| Measure |
Arm I - Modafanil
n=56 participants at risk
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=56 participants at risk
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Injury, poisoning and procedural complications
Fracture
|
0.00%
0/56 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
1.8%
1/56 • Number of events 1 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
Other adverse events
| Measure |
Arm I - Modafanil
n=56 participants at risk
Participants receive modafinil orally (PO) once daily (QD) on days 1-42.
modafinil: Given PO
|
Arm II - Placebo
n=56 participants at risk
Participants receive placebo PO QD on days 1-42.
placebo: Given PO
|
|---|---|---|
|
Nervous system disorders
Headache
|
21.4%
12/56 • Number of events 18 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
12.5%
7/56 • Number of events 9 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
|
Psychiatric disorders
Insomnia
|
23.2%
13/56 • Number of events 16 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
5.4%
3/56 • Number of events 3 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
|
Psychiatric disorders
Agitation
|
7.1%
4/56 • Number of events 4 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
3.6%
2/56 • Number of events 2 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
8.9%
5/56 • Number of events 5 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
0.00%
0/56 • 6 weeks
Complete and frequent monitoring for AEs was incorporated into the study using SAFTEE (Systematic Assessment for Treatment Emergent Effects developed by the National Institute of Mental Health. The final SAFTEE assessment will take place 30 days after the last dose of study agent. Participants will use the Study Medication Log, completed daily, to document any adverse events. Staff will review logs and compare to SAFTEE report.
|
Additional Information
Nicole J. Ullrich, MD, PhD
Children's Hospital Boston/Harvard Medical School
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place