Pharmacokinetics Study of VALTOCO® in Pediatric Subjects With Epilepsy
An Open-Label, Single-Dose, Pharmacokinetics Study of VALTOCO® With Open-Label Safety Period in Pediatric Subjects With Epilepsy
1 other identifier
interventional
36
1 country
13
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Nov 2021
Typical duration for phase_1
13 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 15, 2021
CompletedFirst Posted
Study publicly available on registry
October 13, 2021
CompletedStudy Start
First participant enrolled
November 8, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 11, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
March 11, 2025
CompletedResults Posted
Study results publicly available
September 22, 2026
CompletedSeptember 22, 2026
August 1, 2026
3.3 years
September 15, 2021
August 27, 2026
August 27, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Maximum Plasma Concentration (Cmax) of Diazepam After Intranasal VALTOCO Administered to Subjects With Epilepsy 2 to 5 Years of Age
Pharmacokinetic levels of diazepam
0-6 hours
Study Arms (1)
Valtoco In Pediatric Subjects with Epilepsy
EXPERIMENTAL5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight.
Interventions
5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Neurelis, Inc.lead
Study Sites (13)
Northwest Florida Clinical Research Group, LLC.
Gulf Breeze, Florida, 32561, United States
Nicklaus Children's Hospital
Miami, Florida, 33155, United States
AdventHealth Research Institute
Orlando, Florida, 32803, United States
Center for Rare Neurological Diseases
Norcross, Georgia, 30093, United States
Massachusetts General Hospital
Boston, Massachusetts, 02114, United States
University of Missouri Women & Children's Hospital
Columbia, Missouri, 65201, United States
Northeast Regional Epilepsy Group
Hackensack, New Jersey, 07601, United States
Boston Children's Health Physicians
Hawthorne, New York, 10532, United States
University of Rochester
Rochester, New York, 14623, United States
Duke University Hospital
Durham, North Carolina, 27710, United States
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105, United States
UT Southwestern Medical Center
Dallas, Texas, 75225, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507, United States
Related Publications (2)
Segal EB, Wheless JW, Zafar M, Shih EK, Ngo LY, Carrazana E, Rabinowicz AL; DIAZ.001.008 Study Group. Pharmacokinetics and 180-day safety of diazepam nasal spray in pediatric patients with epilepsy aged 2-5 years. Epilepsia. 2025 Sep;66(9):3231-3241. doi: 10.1111/epi.18473. Epub 2025 Jul 12.
PMID: 40650667RESULTSegal EB, Wheless JW, Wolf SM, Zafar M, Renfroe JB, Lopez-Toledano MA, Ngo LY, Rabinowicz AL, Carrazana E. Safety and Local Tolerability of Diazepam Nasal Spray for Children With Epilepsy Aged 2-5 Years: Final Results of a Phase 1/2a Trial. J Child Neurol. 2026 Aug 26:8830738261474614. doi: 10.1177/08830738261474614. Online ahead of print.
PMID: 42647177DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Miguel Lopez Toledano, PhD., Director
- Organization
- Neurelis, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- No
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 15, 2021
First Posted
October 13, 2021
Study Start
November 8, 2021
Primary Completion
March 11, 2025
Study Completion
March 11, 2025
Last Updated
September 22, 2026
Results First Posted
September 22, 2026
Record last verified: 2026-08