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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

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
Are there results?: Yes, posted in the registrySummary in the doctors' view

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.

City, postcode or hospital

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

Other trials in United States

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 groups in NCT05076838
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.

8 Nov 202111 Mar 2025

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)

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 40650667
Segal 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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Not yet recruiting
Clinical Trial of SEEG for Drug-resistant Epilepsy
Drug-Resistant Epilepsy
RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures
Focal Seizure, Epilepsy
Active, not recruitingObservational study
Transforming Research and Clinical Knowledge in Traumatic Brain Injury Epileptogenesis Project (TRACK-TBI EPI)
Traumatic Brain Injury, Post-traumatic Epilepsy
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1
Focal Seizure, Epilepsy

All Epilepsy trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label, Single-Dose, Pharmacokinetics Study of VALTOCO® With Open-Label Safety Period in Pediatric Subjects With Epilepsy
ClinicalTrials.gov ID: NCT05076838
Acronym / sponsor ID: DIAZ.001.08
Conditions: Epilepsy
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Completed
Enrolment: 36 participants (actual)
Lead sponsor: Neurelis, Inc.Industry (pharma, biotech, devices)
Study start: 8 Nov 2021 (actual)
Primary completion: 11 Mar 2025 (actual)
Study completion: 11 Mar 2025 (actual)
First posted: 13 Oct 2021 (actual)
Results: Posted 22 Sep 2026

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Valtoco In Pediatric Subjects with Epilepsy	Experimental	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.

Who may qualify

Ages: 2 to 5 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

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.

Key exclusion criteria (6 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
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.	Drug	Valtoco In Pediatric Subjects with Epilepsy

Primary outcome

Maximum Plasma Concentration (Cmax) of Diazepam After Intranasal VALTOCO Administered to Subjects With Epilepsy 2 to 5 Years of Age
Time frame: 0-6 hours
Pharmacokinetic levels of diazepam

Results and safety

Results posted to the registry on 22 Sep 2026, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Valtoco In Pediatric Subjects With Epilepsy	36	31

Primary outcome: Maximum Plasma Concentration (Cmax) of Diazepam After Intranasal VALTOCO Administered to Subjects With Epilepsy 2 to 5 Years of Age

Time frame: 0-6 hours. Mean (Standard Deviation). Unit: Cmax (ng/ml)

Measure	Valtoco 5mg 2 to 3 Years Old n = 3	Valtoco 10mg 2 to 3 Years Old n = 15	Valtoco 10mg 4 to 5 Years Old n = 13	Valtoco 15mg 4 to 5 Years Old n = 4
Value	94.34 (60.29)	269.25 (258.77)	279.91 (198.58)	243.07 (249.44)

Adverse events

Time frame: 180 days. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Valtoco 5mg	2 of 3 (66.7%)	3 of 3 (100%)	0 of 3
Valtoco 10mg	6 of 29 (20.7%)	18 of 29 (62.1%)	0 of 29
Valtoco 15mg	2 of 4 (50%)	3 of 4 (75%)	0 of 4

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 40650667Segal 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.
Derived from this study: PubMed 42647177Segal 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.
Link to articleLink provided by the sponsor.

Related trials for Epilepsy

Not yet recruiting
Clinical Trial of SEEG for Drug-resistant Epilepsy
Drug-Resistant Epilepsy
RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures
Focal Seizure, Epilepsy
Active, not recruitingObservational study
Transforming Research and Clinical Knowledge in Traumatic Brain Injury Epileptogenesis Project (TRACK-TBI EPI)
Traumatic Brain Injury, Post-traumatic Epilepsy
RecruitingPhase 3
Study Evaluating the Efficacy and Safety of RAP-219 in Adult Participants With Focal Seizures FOCUS-1
Focal Seizure, Epilepsy

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

13 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Diazepam Nasal Spray [Valtoco] in people with Epilepsy, run by Neurelis, Inc. at 13 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 22 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Maximum Plasma Concentration (Cmax) of Diazepam After Intranasal VALTOCO Administered to Subjects With Epilepsy 2 to 5 Years of Age (time frame: 0-6 hours). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05076838, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Oct 2026.