Narcolepsy Type 1 (NT 1)Phase 2NCT06809803
Extended-release Sodium Oxybate in Children
A Phase 2 study testing Extended-release sodium oxybate and Non-extended-release oxybates in people with Narcolepsy Type 1 (NT 1), run by Stanford University at 1 site in United States.
Trial summary
The main objectives of this study are to determine the treatment preferences of children with narcolepsy type 1 between Extended-release sodium oxybate and Non-extended-release oxybates (Sodium oxybate or a medication with the combination of Calcium, magnesium, potassium, and sodium oxybates). Read more about this study
- Who is it for?
- Ages 7 to 17 years
- Conditions the study is for
- Narcolepsy Type 1 (NT 1)
- What is being tested?
- Extended-release sodium oxybate and Non-extended-release oxybates
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Oct 2025to Jul 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 7 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Participants must be under the care of a doctor at the Stanford Sleep Clinic.
- Participants must have a documented diagnosis of narcolepsy type 1 shown by sleepiness and either: a spinal fluid marker for narcolepsy (hypocretin-1) at a specific level, or a history of sudden loss of muscle control (cataplexy), or a particular genetic marker for narcolepsy, or a sleep study showing a specific sleep pattern for narcolepsy.
- Parent(s), or guardian(s) have signed a consent form and the child must agree to participate.
- Participants are on a stable dose of medications
Who cannot join
- Participants who have any of the following conditions will not be included in the study
- Uncontrolled mental health problems
- Uncontrolled sleep problems that lead to sleepiness.
- Currently having thought about ending one's life or sadness or loss of interest
- Currently having a problem with illegal drug use
- Currently pregnant
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Mila Trabanino contact
United States1 site, 1 recruiting
- Recruiting
Stanford University
Redwood City, California, 94063
Miran Cho, 650-724-4149, mirancho@stanford.edu
Mila Trabanino, milaludi@stanford.edu
About this study
Official title: Safety, Convenience, and Tolerability of a Nightly Single Oral Dose of Extended-release Sodium Oxybate in Children
The main objectives of this study are to determine the treatment preferences of children with narcolepsy type 1 between Extended-release sodium oxybate and Non-extended-release oxybates (Sodium oxybate or a medication with the combination of Calcium, magnesium, potassium, and sodium oxybates). The study will also assess the safety, convenience, and tolerability of Extended-release sodium oxybate. Additionally, the researchers aim to assess how well Extended-release sodium oxybate works in treating narcolepsy type 1.
- Conditions
- Narcolepsy Type 1 (NT 1)
- Being tested
- Extended-release sodium oxybate, Non-extended-release oxybates
- Keywords
- Narcolepsy, Children, Sodium oxybate, treatment
- Medical subject headings
- Narcolepsy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Extended-release sodium oxybateDrug
Also called Lumryz
Participants will take Extended-release sodium Oxybate (Lumryz) 4.5-9 g oral suspension in approximately ⅓ cup of water orally once a day before bedtime. Dosage of Lumryz will be the same as the equivalent total dose of the Non-Extended-release Oxybate (Xywav or Xyrem) that participants use before starting the study
Non-extended-release oxybatesDrug
Also called Xywav, Xyrem
Participants will take Non-Extended-release Oxybates (Xyrem oral solution or Xywav oral solution) 4.5-9 g orally per night (divided into two doses, once before bedtime and another dose 2-4 hours later) at the same dose of Xyrem or Xywav that participants use before starting the study will be continued to compare with Extended-release sodium Oxybate (Lumryz)
| Study group | Role | What they receive |
|---|---|---|
| Extended-release sodium oxybate | Experimental | Participants in this arm will take extended-release sodium oxybate Receives: Drug: Extended-release sodium oxybate; Drug: Non-extended-release oxybates |
| Non-extended-release oxybates | Active comparator | Participants in this arm will take non-extended-release sodium oxybate Receives: Drug: Extended-release sodium oxybate; Drug: Non-extended-release oxybates |
What is being measured
The primary outcome is the main question the study is designed to answer.
Participant preference for selecting the extended vs non-extended release oxybates
Participants will be asked at the end of the study whether participants prefer extended-release sodium oxybate vs sodium oxybate vs calcium, magnesium, potassium and sodium oxybates
Measured: Baseline, up to 8-12 weeks through study completion
Secondary outcomes (3)
Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score
ESS-CHAD is a multi-item questionnaire which asks the participants to rate their chances of falling asleep during various normal daily activities. 4-point Likert scale will be used. The score ranges from 0-24. A higher score means higher chance of falling asleep.
Measured: Baseline, up to 8-12 weeks through study completion
Number of cataplexy attacks
The study team will ask the participants to report the number of cataplexy attacks as measured by Sleep and Symptoms diary when participants switch to the other intervention.
Measured: Baseline, up to 8-12 weeks through study completion
Change in Patient Global Impression of Change (PGIc) scale score
The PGIc is a 7-point scale. Participants will be asked to rate their narcolepsy impression since the end of the Stable-Dose Period. The scale is Very much better, Much better, A little better, No change, A little worse, Much worse, and Very much worse.
Measured: Baseline, up to 8-12 weeks through study completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2025
- Study start27 Oct 2025
- Primary completionJun 2027 (estimated)
- Study completionJul 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Narcolepsy Type 1 (NT 1)Studies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- 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 2
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 36 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Avadel
- Responsible party
- Principal investigator, Oliver Sum-Ping, Stanford University
- Investigators
- Oliver Sum-Ping, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06809803
- Sponsor's protocol ID
- 75896
- Status last verified
- Nov 2025
- First submitted
- 30 Jan 2025
- Last updated
- 6 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety, Convenience, and Tolerability of a Nightly Single Oral Dose of Extended-release Sodium Oxybate in Children
- ClinicalTrials.gov ID
- NCT06809803
- Acronym / sponsor ID
- 75896
- Conditions
- Narcolepsy Type 1 (NT 1)
- Keywords
- Narcolepsy, Children, Sodium oxybate, treatment
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 36 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Avadel
- Study start
- 27 Oct 2025 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jul 2027 (estimated)
- First posted
- 5 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objectives of this study are to determine the treatment preferences of children with narcolepsy type 1 between Extended-release sodium oxybate and Non-extended-release oxybates (Sodium oxybate or a medication with the combination of Calcium, magnesium, potassium, and sodium oxybates). The study will also assess the safety, convenience, and tolerability of Extended-release sodium oxybate. Additionally, the researchers aim to assess how well Extended-release sodium oxybate works in treating narcolepsy type 1.
Study design
- Primary purpose
- Other
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Extended-release sodium oxybate | Experimental | Drug: Extended-release sodium oxybate, Drug: Non-extended-release oxybatesParticipants in this arm will take extended-release sodium oxybate |
| Non-extended-release oxybates | Active comparator | Drug: Extended-release sodium oxybate, Drug: Non-extended-release oxybatesParticipants in this arm will take non-extended-release sodium oxybate |
Who may qualify
- Ages
- 7 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Participants must be under the care of a doctor at the Stanford Sleep Clinic.
- Participants must have a documented diagnosis of narcolepsy type 1 shown by sleepiness and either: a spinal fluid marker for narcolepsy (hypocretin-1) at a specific level, or a history of sudden loss of muscle control (cataplexy), or a particular genetic marker for narcolepsy, or a sleep study showing a specific sleep pattern for narcolepsy.
- Parent(s), or guardian(s) have signed a consent form and the child must agree to participate.
- Participants are on a stable dose of medications
Key exclusion criteria (6 listed)
- Participants who have any of the following conditions will not be included in the study
- Uncontrolled mental health problems
- Uncontrolled sleep problems that lead to sleepiness.
- Currently having thought about ending one's life or sadness or loss of interest
- Currently having a problem with illegal drug use
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 |
|---|---|---|
| Extended-release sodium oxybateAlso: LumryzParticipants will take Extended-release sodium Oxybate (Lumryz) 4.5-9 g oral suspension in approximately ⅓ cup of water orally once a day before bedtime. Dosage of Lumryz will be the same as the equivalent total dose of the Non-Extended-release Oxybate (Xywav or Xyrem) that participants use before starting the study | Drug | Extended-release sodium oxybate, Non-extended-release oxybates |
| Non-extended-release oxybatesAlso: Xywav, XyremParticipants will take Non-Extended-release Oxybates (Xyrem oral solution or Xywav oral solution) 4.5-9 g orally per night (divided into two doses, once before bedtime and another dose 2-4 hours later) at the same dose of Xyrem or Xywav that participants use before starting the study will be continued to compare with Extended-release sodium Oxybate (Lumryz) | Drug | Extended-release sodium oxybate, Non-extended-release oxybates |
Primary outcome
Participant preference for selecting the extended vs non-extended release oxybates
Time frame: Baseline, up to 8-12 weeks through study completion
Participants will be asked at the end of the study whether participants prefer extended-release sodium oxybate vs sodium oxybate vs calcium, magnesium, potassium and sodium oxybates
3 secondary outcomes
Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score
Time frame: Baseline, up to 8-12 weeks through study completion
Number of cataplexy attacks
Time frame: Baseline, up to 8-12 weeks through study completion
Change in Patient Global Impression of Change (PGIc) scale score
Time frame: Baseline, up to 8-12 weeks through study completion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2025.
- Individual participant dataSharing planned: No
Related trials for Narcolepsy Type 1 (NT 1)
Active, not recruitingObservational study
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Narcolepsy Type 1 (NT 1), Hypersomnolence Disorder
Recruiting
A Novel Approach to Manage Symptoms of Narcolepsy and Idiopathic Hypersomnia
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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.
Mila Trabanino contact
Oliver Sum-Ping, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Extended-release sodium oxybate and Non-extended-release oxybates in people with Narcolepsy Type 1 (NT 1), run by Stanford University at 1 site 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 4 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: looking for participants now, as of the sponsor's last update on 6 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 phase mean?
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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Miran Cho (650-724-4149, mirancho@stanford.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Participant preference for selecting the extended vs non-extended release oxybates (time frame: Baseline, up to 8-12 weeks through study completion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 6 Nov 2025.