Epilepsy, Anxiety
Conditions
Keywords
pediatrics
Brief summary
This study is an open label, adjunctive, proof of concept, pilot clinical trial. Pediatric patients with epilepsy and clinically significant anxiety will be recruited and if enrolled will receive active treatment, involving flexible dose titration of clobazam and will be monitored for a period of four months. The study will be monitored and overseen by the Johns Hopkins Hospital Institutional Review Board.
Interventions
DRUGClobazam
Clobazam is used as an adjunct medicine for all participants.
Sponsors
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
All participants receive treatment and outcome is assessed; there is no control group.
Eligibility
Sex/Gender
ALL
Age
6 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Established diagnosis of epilepsy, characterized by focal seizures with suspected or documented localization in the temporal lobe. All participants will have active epilepsy that requires treatment with anticonvulsant medication. * Although it is not necessary to be seizure free, a seizure baseline period will be established in the 60 days prior to enrollment into the study. * Current regimen of anticonvulsant drugs must have been stable for 30 days prior to entry into the study. * No episodes of seizure clusters of status epilepticus within 30 days prior to entry into the study. * Established symptoms of anxiety with functional impairment. * A diagnosis of an anxiety disorder based on the administration of the K-SADS Male or female participants equal to or above age 6 and below age 18 at the start of the study. No exclusion will be made on the basis of gender or minority status. * Male or female participants equal to or above age 6 and below age 18 at the start of the study. No exclusion will be made on the basis of gender or minority status. * Good general health as determined by medical history and physical examination. * Ability to swallow pills (participant will receive pill swallowing instruction if necessary). The medicine may be cut into pieces and/or mixed with applesauce. * If female of childbearing age, a negative urine or serum pregnancy test must be established or assured at baseline. Additionally, the participant must agree to use abstinence or appropriate contraception methods or be otherwise incapable of pregnancy for the duration of the study. Pregnancy test results will be shared with parent or guardian. Pregnancy status (or prevention) and abstinence or contraception methods will be addressed throughout the study for females of childbearing age as well as for post-pubertal males. * Previous subjects who failed at any point to meet continuation criteria and withdrew early may be considered for re-enrollment by the PI on a case-by-case basis. * Participant or legal caregiver capable of providing informed consent and fully capable of monitoring the subject's disease process and compliance with treatment.
Exclusion criteria
* Previous allergic or hypersensitivity reactions to Onfi® or benzodiazepines * Active substance abuse or dependence within 30 days of enrollment * DSM-V diagnosis of psychotic illness or imminent risk of harm to self or others. * Current standing use of benzodiazepines (except as rescue medicine) * Serious or unstable medical or neurologic conditions such as HIV, liver or kidney disease, cancer or diabetes. * Participation in a previous experimental drug study within 30 days of baseline visit. * Estimated IQ\<70 as indicated by initial clinical assessment (rendering rating scales invalid) * Insufficient capacity of caregiver or legal guardian to understand and appropriately consent for study procedures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| CGI-I | 14-18 weeks | Mean of Clinical Global Impression-Improvement from baseline. (1-7 point scale; lower score equals a better outcome; 1 is the maximum improvement, 7 is the minimum improvement) |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Open Label Single Treatment Arm open label
Clobazam: Clobazam is used as an adjunct medicine for all participants. | 20 |
| Total | 20 |
Baseline characteristics
| Characteristic | Open Label Single Treatment Arm |
|---|---|
| Age, Categorical <=18 years | 20 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Age, Continuous | 13.8 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 5 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 14 Participants |
| Region of Enrollment United States | 20 Participants |
| Sex: Female, Male Female | 16 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 20 |
| other Total, other adverse events | 0 / 20 |
| serious Total, serious adverse events | 0 / 20 |
Outcome results
Primary
CGI-I
Mean of Clinical Global Impression-Improvement from baseline. (1-7 point scale; lower score equals a better outcome; 1 is the maximum improvement, 7 is the minimum improvement)
Time frame: 14-18 weeks
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Clobazam | CGI-I | 2 units on a scale |