Clinical trial NCT05076838: Pharmacokinetics Study of VALTOCO® in Pediatric Subjects With Epilepsy
Official title: An Open-Label, Single-Dose, Pharmacokinetics Study of VALTOCO® With Open-Label Safety Period in Pediatric Subjects With Epilepsy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Epilepsy
Lead sponsor: Neurelis, Inc.
Sponsor class: INDUSTRY
First posted: 2021-10-13
Enrollment: 35
Sex: ALL
Minimum age (years): 2
Maximum age (years): 5
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a Phase 1/2a, open-label, single-dose, PK study of VALTOCO with open-label safety period in pediatric epilepsy subjects age 2 to 5 years.


Eligibility criteria: Inclusion Criteria:

* Subject has a clinical diagnosis of epilepsy and, in the opinion of the Investigator, may need benzodiazepine intervention for seizure control.
* Subjects having either partial or generalized epilepsy with motor seizures or seizures with clear alteration of awareness, for which rescue medications have been used at least once in the last 3 months or in the opinion of the investigator, may need a benzodiazepine intervention for seizure control 1-2 times every 3 months on average.

Exclusion Criteria:

* Subjects whose body weight are \< 6 kg or \> 33 kg.
* Subject is undergoing intracranial electroencephalogram (EEG) monitoring.
* In the opinion of the investigator, a history of clinically significant medical history that would jeopardize the safety of the subject or impact the validity of the study results (such as significant gastrointestinal, renal, nasal polyps or any nasal passage abnormality that could interfere with nasal spray administration.
* Subject has had significant traumatic injury, major surgery or open biopsy within 30 days prior to study screening.
* Participation in a clinical trial within 30 days prior to the first dose of study drug.
* Inadequate or difficult venous access that may jeopardize the quality or timing of the PK samples.

Primary outcomes:
measure: Maximum Plasma Concentration (Cmax) of diazepam after intranasal VALTOCO administered to subjects with epilepsy 2 to 5 years of age
timeFrame: 0-6 hours
Secondary outcomes:
Other outcomes:
Locations:
city: Gulf Breeze
country: United States
facility: Northwest Florida Clinical Research Group, LLC.
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Orlando
country: United States
facility: AdventHealth Research Institute
state: Florida
city: Norcross
country: United States
facility: Center for Rare Neurological Diseases
state: Georgia
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Columbia
country: United States
facility: University of Missouri Women & Children's Hospital
state: Missouri
city: Hackensack
country: United States
facility: Northeast Regional Epilepsy Group
state: New Jersey
city: Hawthorne
country: United States
facility: Boston Children's Health Physicians
state: New York
city: Rochester
country: United States
facility: University of Rochester
state: New York
city: Durham
country: United States
facility: Duke University Hospital
state: North Carolina
city: Memphis
country: United States
facility: Le Bonheur Children's Hospital
state: Tennessee
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Norfolk
country: United States
facility: Children's Hospital of the King's Daughters
state: Virginia
Study officials:
References: