EpilepsyPhase 1/2NCT05076838
Pharmacokinetics Study of VALTOCO® in Pediatric Subjects With Epilepsy
A Phase 1/2 study testing Diazepam Nasal Spray [Valtoco] in people with Epilepsy, run by Neurelis, Inc. at 13 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 5 years
- Conditions the study is for
- Epilepsy
- What is being tested?
- Diazepam Nasal Spray [Valtoco]
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 13 sitesUnited States
- Study dates
- 8 Nov 2021to 11 Mar 2025
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
United States13 sites
Massachusetts General Hospital
Boston, Massachusetts, 02114
University of Missouri Women & Children's Hospital
Columbia, Missouri, 65201
UT Southwestern Medical Center
Dallas, Texas, 75225
Duke University Hospital
Durham, North Carolina, 27710
Northwest Florida Clinical Research Group, LLC.
Gulf Breeze, Florida, 32561
Northeast Regional Epilepsy Group
Hackensack, New Jersey, 07601
Boston Children's Health Physicians
Hawthorne, New York, 10532
Le Bonheur Children's Hospital
Memphis, Tennessee, 38105
Nicklaus Children's Hospital
Miami, Florida, 33155
Center for Rare Neurological Diseases
Norcross, Georgia, 30093
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
AdventHealth Research Institute
Orlando, Florida, 32803
University of Rochester
Rochester, New York, 14623
About this study
Official title: 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.
- Conditions
- Epilepsy
- Being tested
- Diazepam Nasal Spray [Valtoco]
- Medical subject headings
- Epilepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Diazepam Nasal Spray [Valtoco]Drug
5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight.
| Study group | Role | What they receive |
|---|---|---|
| Valtoco In Pediatric Subjects with Epilepsy | Experimental | 5 mg, 10 mg, or 15 mg dose of intranasal VALTOCO will be administered based on the subject's body weight. Receives: Drug: Diazepam Nasal Spray [Valtoco] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Plasma Concentration (Cmax) of Diazepam After Intranasal VALTOCO Administered to Subjects With Epilepsy 2 to 5 Years of Age
Pharmacokinetic levels of diazepam
Measured: 0-6 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Oct 2021
- Study start8 Nov 2021
- Primary completion11 Mar 2025
- Study completion11 Mar 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for EpilepsyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Neurelis, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05076838
- Sponsor's protocol ID
- DIAZ.001.08
- Status last verified
- Sep 2026
- First submitted
- 15 Sep 2021
- Last updated
- 20 Oct 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40650667Segal 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.
PubMed 42647177