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A Phase II Placebo-Controlled Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Brain Tumor

Phase 2
6 Years
17 Years
Open (Enrolling)
Brain and Central Nervous System Tumors, Cognitive/Functional Effects, Fatigue, Neurotoxicity, Psychosocial Effects of Cancer and Its Treatment

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Trial Information

A Phase II Placebo-Controlled Trial of Modafinil to Improve Neurocognitive Deficits in Children Treated for a Primary Brain Tumor



- Determine whether a 6-week drug trial of modafinil, compared to placebo, is associated
with improvement in neurocognitive function as defined by parent report of inattention
or working memory deficits or by direct assessment of attention, working memory, or
processing speed in children with cognitive impairment after treatment for a primary
brain tumor.


- Determine whether modafinil, compared to placebo, is associated with improved executive
function (apart from working memory), as assessed using the BRIEF executive function
and hippocampal learning and executive function tasks from the CogState battery.

- Determine whether modafinil, compared to placebo, is associated with reduced fatigue as
assessed using the PedsQL Multidimensional Fatigue Scale.

- Evaluate the safety of modafinil in this population.

OUTLINE: This is a multicenter study. Participants are randomized to 1 of 2 treatment arms.

- Arm I: Participants receive modafinil orally (PO) once daily (QD) on days 1-42.

- Arm II: Participants receive placebo PO QD on days 1-42. Participants complete a
semi-automated, computerized cognitive-testing system (CogState) designed to assess
psychomotor, attention/vigilance, memory, and other components of executive function by
presenting different tasks, each with its own set of rules, at baseline and after
completion of study therapy. Participants also complete the PedsQL Multidimensional
Fatigue Scale (Peds QL-MFS).

Parents or legal guardians complete the PedsQL-MFS, the Conners Parent Reported Scale
(CPR-3), and the Behavior Rating Inventory of Executive Function (BRIEF) at baseline and
after completion of study therapy.

Clinical and/or research staff administer the Systematic Assessment for Treatment Emergency
Events (SAFTEE), a semi-structured interview designed to elicit adverse events, at baseline
and periodically during study.

After completion of study therapy, participants are followed up for 30 days.

Inclusion Criteria


- Age ≥ 6 years and ≤ 17 years 10 months at the time of study entry (so that
participants will be < 18 at the 6 week evaluation, which is the upper age limit for
which the included instruments are valid).

- Diagnosis of a primary brain tumor treated with at least one of the following:

1. neurosurgical resection of the brain tumor;

2. cranial irradiation; or

3. any chemotherapy to treat the brain tumor.

- Off-treatment and progression-free for at least 12 months and ≤ 84 months. Treatment
cessation is defined as the final dose of chemotherapy, the last dose (fraction) of
radiation or date of surgery, whichever occurred last.

- Parent/Legal Guardian and child able to read English.

- Vision and hearing (eyeglasses and/or hearing aid permissible) sufficient for valid
test administration and cooperation with examinations.

- Availability of a reliable parent or legal guardian who is willing and able to
complete all of the outcome measures and fulfill the requirements of the study,
including administration of medications and accompanying the participant to all study

- Females of childbearing potential must have a negative pregnancy test result and must
agree to use a medically acceptable method of contraception throughout the entire
study period and for 30 days after the last dose of study drug.

- Childbearing potential is defined as girls who are >Tanner stage 2, except for those
who have documented pan pituitary insufficiency or other hormonal state incompatible
with pregnancy.

- Urine pregnancy tests are acceptable.


- Off treatment > 84 months

- Inability to perform the testing procedure (for example, because of aphasia, motor
deficits affecting the dominant hand, or IQ < 70)

- Known cardiac disorders including arrhythmias, hypertension requiring treatment or
structural heart disease

- Diagnosis of narcolepsy, sick sinus syndrome, arrhythmia or prolonged QTc

- History of stroke or head injury associated with loss of consciousness within 12
months of registration

- History of grade 2 depression or anxiety or treatment with antidepressants,
antipsychotics or MAO inhibitors within 30 days of registration

- Concurrent treatment with any medications or substances that are potent inhibitors or
inducers of CYP3A4, hepatic enzyme inducing antiepileptic drugs (EIAEDs),or other
drugs known to affect the metabolism of modafinil. Examples include but are not
limited to itraconazole, ketoconazole, doxycycline, rifampin, St. John's wort,
phenytoin, phenobarbital, diazepam, tricyclic antidepressants.

- If patients were previously taking, EIAEDs, they must be off for > 2 weeks prior to
study enrollment.

- Treatment with other stimulant medications within 14 days of registration; however, a
diagnosis of ADHD does NOT exclude a child from participation

- Participants with known hypersensitivity to modafinil, armodafinil or any of its

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Supportive Care

Outcome Measure:

Change in age-adjusted scores at week six from baseline in any of the 5 questionnaires

Outcome Time Frame:

6 weeks

Safety Issue:


Principal Investigator

Jeffrey P. Krischer, MD, PhD

Investigator Role:

Study Chair

Investigator Affiliation:

University of South Florida


United States: Data and Safety Monitoring Board

Study ID:

SCUSF 0901



Start Date:

August 2011

Completion Date:

Related Keywords:

  • Brain and Central Nervous System Tumors
  • Cognitive/Functional Effects
  • Fatigue
  • Neurotoxicity
  • Psychosocial Effects of Cancer and Its Treatment
  • childhood brain tumor
  • neurotoxicity
  • fatigue
  • cognitive/functional effects
  • psychosocial effects of cancer and its treatment
  • Brain Neoplasms
  • Fatigue
  • Nervous System Neoplasms
  • Central Nervous System Neoplasms
  • Neurotoxicity Syndromes



Mayo Clinic Rochester, Minnesota  55905
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
University of Texas Health Science Center at San Antonio San Antonio, Texas  78284-7811
MD Anderson Cancer Center Orlando Orlando, Florida  32806
St. Vincent Hospital Green Bay, Wisconsin  54307-3508
Children's Hospital Los Angeles Los Angeles, California  90027-0700
Children's National Medical Center Washington, District of Columbia  20010-2970
All Children's Hospital St. Petersburg, Florida  33701
University of Oklahoma Health Sciences Center Oklahoma City, Oklahoma  73104
St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794
Driscoll Children's Hospital Corpus Christi, Texas  78466
University of Illinois at Chicago Chicago, Illinois  60612
Kosair Children's Hospital Louisville, Kentucky  40202-3830
East Tennessee Children's Hospital Knoxville, Tennessee  37901
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas Dallas, Texas  75390
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center New York, New York  10032
Vanderbilt Children's Hospital Nashville, Tennessee  37232-6310
Saint Louis University Cancer Center Saint Louis, Missouri  63110
University of Alabama at Birmingham Birmingham, Alabama  35294-3300
Children's Hospital Boston Boston, Massachusetts  02115
Connecticut Children's Medical Center Hartford, Connecticut  06106
University of California San Francisco San Francisco, California  941104206
Kapiolani Medical Center for Women and Children Honolulu, Hawaii  96826
SUNY Upstate Medical University Syracuse, New York  13210
University of New Mexico Albuquerque, New Mexico  87131
Children's Healthcare of Atlanta Atlanta, Georgia  30342
University of Mississippi Jackson, Mississippi  39216
Children's Mercy Hospital and Clinics Kansas City, Missouri  64108
SunCoast CCOP Research Base at the University of South Florida Tampa, Florida  33612
Lucile Packard Children's Hospital Stanford University Palo Alto, California  94304
Children's Hospital of Colorado; Saint Joseph Hospital Denver, Colorado  80218
Nemours Children's Clinic- Pensacola Pensacola, Florida  32207
Riley Hospital for Children- Indiana University Indianapolis, Indiana  46163
CS Mott/University of Michigan Ann Arbor, Michigan  48109
Doernbecher Children's Hospital/ Oregoon Health Science University Portland, Oregon  97329