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

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.

Study Overview

• Status: Completed
• Conditions: Epilepsy
• Intervention / Treatment: Drug: Diazepam Nasal Spray [Valtoco]

• Study Type: Interventional
• Enrollment (Actual): 35
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Gulf Breeze, Florida, United States, 32561
      • Northwest Florida Clinical Research Group, LLC.
    • Miami, Florida, United States, 33155
      • Nicklaus Children's Hospital
    • Orlando, Florida, United States, 32803
      • AdventHealth Research Institute
  • Georgia
    • Norcross, Georgia, United States, 30093
      • Center for Rare Neurological Diseases
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Massachusetts General Hospital
  • Missouri
    • Columbia, Missouri, United States, 65201
      • University of Missouri Women & Children's Hospital
  • New Jersey
    • Hackensack, New Jersey, United States, 07601
      • Northeast Regional Epilepsy Group
  • New York
    • Hawthorne, New York, United States, 10532
      • Boston Children's Health Physicians
    • Rochester, New York, United States, 14623
      • University of Rochester
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Hospital
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Le Bonheur Children's Hospital
  • Texas
    • Dallas, Texas, United States, 75225
      • UT Southwestern Medical Center
  • Virginia
    • Norfolk, Virginia, United States, 23507
      • Children's Hospital of The King's Daughters

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 5 years (Child)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Valtoco In Pediatric Subjects with Epilepsy; 5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight.	Drug: Diazepam Nasal Spray [Valtoco]; 5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Maximum Plasma Concentration (Cmax) of diazepam after intranasal VALTOCO administered to subjects with epilepsy 2 to 5 years of age	0-6 hours

Collaborators and Investigators

• Sponsor: Neurelis, Inc.

Study record dates

Study Major Dates

• Study Start (Actual): November 8, 2021
• Primary Completion (Actual): March 11, 2025
• Study Completion (Actual): March 11, 2025

Study Registration Dates

• First Submitted: September 15, 2021
• First Submitted That Met QC Criteria: October 12, 2021
• First Posted (Actual): October 13, 2021

Study Record Updates

• Last Update Posted (Actual): August 11, 2025
• Last Update Submitted That Met QC Criteria: August 5, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Antiemetics; Autonomic Agents; Peripheral Nervous System Agents; Gastrointestinal Agents; Anesthetics; Central Nervous System Depressants; Neurotransmitter Agents; Adjuvants, Anesthesia; Hypnotics and Sedatives; Anti-Anxiety Agents; Tranquilizing Agents; Psychotropic Drugs; Anesthetics, Intravenous; Anesthetics, General; GABA Modulators; GABA Agents; Anticonvulsants; Neuromuscular Agents; Muscle Relaxants, Central; Diazepam
• Other Study ID Numbers: DIAZ.001.08

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No