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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Clobazam | Other | open label (single treatment arm) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Clobazam | Drug | Clobazam is used as an adjunct medicine for all participants. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | 14-18 weeks |
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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.
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:
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| Name | Affiliation | Role |
|---|---|---|
| Jay Salpekar, MD | KKI/JH | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Kennedy Krieger Institute | Baltimore | Maryland | 21205 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Clobazam | open label single treatment arm Clobazam is used as an adjunct medicine for all participants. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Open Label Single Treatment Arm | open label Clobazam: Clobazam is used as an adjunct medicine for all participants. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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) | Posted | Mean | Full Range | units on a scale | 14-18 weeks |
|
|
up to 18 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Open Label Single Treatment Arm | open label Clobazam: Clobazam is used as an adjunct medicine for all participants. |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Project Director | KennedyKrieger | 443-923-7600 | Salpekar@kennedykrieger.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Dec 12, 2017 | Dec 12, 2017 | Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D004827 | Epilepsy |
| D001008 | Anxiety Disorders |
| ID | Term |
|---|---|
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D001523 | Mental Disorders |
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| ID | Term |
|---|---|
| D000078306 | Clobazam |
| ID | Term |
|---|---|
| D001569 | Benzodiazepines |
| D001552 | Benzazepines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
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All participants receive treatment and outcome is assessed; there is no control group.
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| Participants |
|
| Age, Continuous | Mean | Full Range | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Count of Participants | Participants |
|
|
| 0 |
| 20 |
| 0 |
| 20 |
| 0 |
| 20 |
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| D006571 | Heterocyclic Compounds |