36 Participants Needed

Extended-release Sodium Oxybate for Narcolepsy

SL
MC
Overseen ByMiran Cho
Age: < 18
Sex: Any
Trial Phase: Phase 2
Sponsor: Stanford University
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial requires participants to be on a stable dose of their current medications, so you will not have to stop taking them.

Is extended-release sodium oxybate safe for humans?

Extended-release sodium oxybate is under review for treating narcolepsy, and while specific long-term safety data for this formulation is limited, sodium oxybate has been used for narcolepsy for many years. Safety concerns with immediate-release versions include dosing errors, but no specific safety issues for the extended-release version have been highlighted in the available research.12345

How is the drug extended-release sodium oxybate different from other narcolepsy treatments?

Extended-release sodium oxybate is unique because it is a once-nightly formulation, unlike other sodium oxybate treatments that require waking up in the middle of the night for a second dose. This makes it more convenient and reduces the risk of dosing errors.12567

Research Team

OS

Oliver Sum-Ping, MD

Principal Investigator

Stanford University

Eligibility Criteria

This trial is for children with narcolepsy type 1, which includes those who experience sudden muscle weakness (cataplexy). Specific eligibility details are not provided, but typically participants must meet certain health standards and not be taking conflicting medications.

Inclusion Criteria

Participants must be under the care of a doctor at the Stanford Sleep Clinic
I have narcolepsy type 1, confirmed by tests or symptoms.
Parent(s), or guardian(s) have signed a consent form and the child must agree to participate
See 1 more

Exclusion Criteria

I have sleep problems that make me very sleepy.
Participants who are currently pregnant
Participants who have uncontrolled mental health problems
See 2 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either extended-release sodium oxybate or non-extended-release oxybates to assess treatment preferences, safety, convenience, and tolerability

8-12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

  • Extended-release Sodium Oxybate
Trial OverviewThe study compares children's preferences between traditional sodium oxybate treatments and a new extended-release version. It also evaluates the safety, convenience, and effectiveness of the extended-release medication in treating narcolepsy type 1.
Participant Groups
2Treatment groups
Experimental Treatment
Active Control
Group I: Extended-release sodium oxybateExperimental Treatment2 Interventions
Participants in this arm will take extended-release sodium oxybate
Group II: Non-extended-release oxybatesActive Control2 Interventions
Participants in this arm will take non-extended-release sodium oxybate

Find a Clinic Near You

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials
2,527
Recruited
17,430,000+

Avadel

Industry Sponsor

Trials
5
Recruited
660+

Findings from Research

In a long-term study involving 201 participants with narcolepsy and cataplexy, low-sodium oxybate (LXB) demonstrated a safety profile similar to that of sodium oxybate (SXB), with common treatment-emergent adverse events (TEAEs) like headache, nausea, and dizziness occurring mostly early in treatment and generally lasting a short duration.
The prevalence of TEAEs decreased over the course of the 24-week open-label extension, indicating that LXB is well-tolerated over time, with headache being the most frequently reported adverse event during both the main study and the extension period.
Long-Term Safety and Tolerability During a Clinical Trial and Open-Label Extension of Low-Sodium Oxybate in Participants with Narcolepsy with Cataplexy.Bogan, RK., Foldvary-Schaefer, N., Skowronski, R., et al.[2023]
In a study of 90 patients with severe narcolepsy and cataplexy who were resistant to other treatments, sodium oxybate significantly reduced daytime sleepiness and weekly cataplexy events, indicating its efficacy in clinical practice.
While sodium oxybate was effective, about 26.6% of patients had to stop treatment due to side effects, with older patients being more susceptible to serious side effects like psychosis.
Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice.Drakatos, P., Lykouras, D., D'Ancona, G., et al.[2021]
Sodium oxybate (Xyrem) is the only FDA-approved treatment specifically for cataplexy in narcolepsy and has recently been approved for excessive daytime sleepiness associated with the condition.
Unlike traditional antidepressants and stimulants, sodium oxybate effectively addresses both cataplexy and daytime sleepiness with a lower risk of tolerance and side effects when used correctly.
Sodium oxybate for narcolepsy.Scharf, MB.[2015]

References

Evidence of Accidental Dosing Errors with Immediate-Release Sodium Oxybate: Data from the US Food and Drug Administration Adverse Event Reporting System. [2023]
Clinician Preferences for Oxybate Treatment for Narcolepsy: Survey and Discrete Choice Experiment. [2023]
Long-Term Safety and Tolerability During a Clinical Trial and Open-Label Extension of Low-Sodium Oxybate in Participants with Narcolepsy with Cataplexy. [2023]
Safety and efficacy of long-term use of sodium oxybate for narcolepsy with cataplexy in routine clinical practice. [2021]
Effect of FT218, a Once-Nightly Sodium Oxybate Formulation, on Disrupted Nighttime Sleep in Patients with Narcolepsy: Results from the Randomized Phase III REST-ON Trial. [2022]
Efficacy of once-nightly sodium oxybate (FT218) in narcolepsy type 1 and type 2: post hoc analysis from the Phase 3 REST-ON Trial. [2023]
Sodium oxybate for narcolepsy. [2015]