Extended-release Sodium Oxybate for Narcolepsy

Overseen By Mila Trabanino

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Stanford University

Disqualifiers: Uncontrolled mental health, sleep problems, illegal drug use, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 8-12 weeks

36 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine which treatment children with narcolepsy type 1 prefer: extended-release sodium oxybate or non-extended-release versions. It will also assess the safety, convenience, and effectiveness of the extended-release form for treating narcolepsy type 1. Children diagnosed with narcolepsy type 1 and receiving care at the Stanford Sleep Clinic may qualify for the trial. Participants must already be on a stable medication dose and have a documented diagnosis of narcolepsy type 1. The trial involves taking either the new extended-release treatment or the standard non-extended-release option. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, allowing participants to contribute to significant findings.

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 there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that extended-release sodium oxybate is usually well-tolerated. In past studies, most participants did not discontinue the medication due to side effects. About 6% of those using a similar immediate-release version experienced sleepwalking, but none stopped treatment because of it. Long-term safety data indicates that no new safety issues have emerged with this treatment, suggesting it is relatively safe for most people. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for narcolepsy?

Unlike the standard treatments for narcolepsy that usually involve immediate-release formulations requiring multiple doses, extended-release sodium oxybate is designed to be taken less frequently. This extended-release formula provides a more consistent delivery of the medication throughout the night, potentially leading to improved sleep quality and reduced daytime sleepiness. Researchers are excited about this treatment because it could simplify medication regimens for patients, enhance adherence, and offer more stable symptom control compared to non-extended-release oxybates.

What evidence suggests that extended-release sodium oxybate might be an effective treatment for narcolepsy?

Studies have shown that extended-release sodium oxybate, which participants in this trial may receive, can greatly improve narcolepsy symptoms. It particularly helps with excessive daytime sleepiness and cataplexy, a sudden muscle weakness. Research indicates that individuals taking this once-nightly medication adhere better to their treatment plan. This version of the medication also enhances daytime functioning compared to the non-extended-release version, which participants in another arm of this trial will take. Overall, it offers a more convenient and effective way to manage narcolepsy symptoms. ^{2 6 7 8 9}

Who Is on the Research Team?

Oliver Sum-Ping, MD

Principal Investigator

Stanford University

Are You a Good Fit for This Trial?

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

Exclusion Criteria

I have sleep problems that make me very sleepy.

Participants who are currently pregnant

Participants who have uncontrolled mental health problems

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

• Extended-release Sodium Oxybate

Trial Overview The 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.

How Is the Trial Designed?

Group I: Extended-release sodium oxybate Experimental Treatment 2 Interventions

Participants in this arm will take extended-release sodium oxybate

Group II: Non-extended-release oxybates Active Control 2 Interventions

Participants in this arm will take non-extended-release sodium oxybate

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials

2,527

Recruited

17,430,000+

Avadel

Industry Sponsor

Trials

5

Recruited

660+

Published Research Related to This Trial

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sodium oxybate for narcolepsy. Scharf, MB.[2015]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11107209/

Analysis from the phase 3 REST-ON clinical trial - PMC Improvement in sleep latency with extended-release once-nightly sodium oxybate for the treatment of adults with narcolepsy: Analysis from the phase 3 REST-ON ...

2. investors.avadel.com investors.avadel.com/news-releases/news-release-details/avadel-pharmaceuticals-present-new-data-lumryztm-sodium-0

Avadel Pharmaceuticals to Present New Data on LUMRYZ ... LUMRYZ is an extended-release sodium oxybate medication approved by the FDA on May 1, 2023 , as the first and only once-at-bedtime treatment for ...

3. neurologylive.com neurologylive.com/view/real-world-data-benefits-once-nightly-oxybate-narcolepsy-management

Real-World Data on the Benefits of Once-Nightly Oxybate ... Both experts noted significant improvements in patient adherence and daytime functioning compared with twice-nightly dosing, as well as reduced ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S138994572400443X

Efficacy of once-nightly sodium oxybate (FT218) on ... Extended-release, once-nightly sodium oxybate (ON-SXB) significantly improved narcolepsy symptoms in participants in the phase 3, randomized, double-blind, ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT02720744

NCT02720744 | Once-Nightly Sodium Oxybate for ... The purpose of this study is to determine whether once-nightly FT218 is safe and effective for the treatment of excessive daytime sleepiness and cataplexy in ...

6. lumryzhcp.com lumryzhcp.com/clinical-results/lumryz-safety

Safety | LUMRYZ™ (sodium oxybate) for extended-release ... Learn about the safety and tolerability of LUMRYZ™. See full Prescribing Information, including BOXED Warning, and Medication Guide.

7. neurology.org neurology.org/doi/10.1212/WNL.0000000000201923

Long-Term Safety of Once-Nightly Sodium Oxybate Thus far in RESTORE, ON-SXB is generally well tolerated, and no new safety signals have been observed. If approved, ON-SXB will offer a once-nightly oxybate ...

8. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK562283/

Sodium Oxybate - StatPearls - NCBI Bookshelf Sodium oxybate is a medication that helps manage and treat narcolepsy with cataplexy or excessive daytime sleepiness.

9. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2023/214755Orig1s000lbl.pdf

Lumryz (sodium oxybate) - accessdata.fda.gov No patients treated with LUMRYZ discontinued due to sleepwalking. Sleepwalking was reported in 6% of 781 patients with narcolepsy treated with immediate- ...