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oxybate

Phase 2

Narcolepsy Type 1 (NT 1) | Small molecule | Neurology |Avadel Pharmaceuticals plc|Last Updated: Nov 6, 2025

Target and mechanism

Molecular targetGABBR1, GABBR2
Target classAgonist
ModalitySmall molecule

Also known as Extended-release sodium oxybate

Success Probability

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Market & Valuation

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Trial Design

RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment36

FDA Designations

No designations recorded

Clinical trial landscape

oxybate · 1 trial · 1 indication

Phase 2 1
NCT06809803Extended-release Sodium Oxybate in ChildrenNarcolepsy Type 1 (NT 1)
RECRUITING36 Analytics
PHASE2RECRUITING
Extended-release Sodium Oxybate in Children
Narcolepsy Type 1 (NT 1)Unlock trial analytics

Study Endpoints

Primary Endpoints

Participant preference for selecting the extended vs non-extended release oxybates
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

Secondary Endpoints

Change in Epworth Sleepiness Scales for Children and Adolescents (ESS-CHAD) score
Baseline, up to 8-12 weeks through study completion
Number of cataplexy attacks
Baseline, up to 8-12 weeks through study completion
Change in Patient Global Impression of Change (PGIc) scale score
Baseline, up to 8-12 weeks through study completion
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelCROSSOVER
PurposeOTHER

Treatment Arms

ArmTypeDescription
Extended-release sodium oxybateEXPERIMENTALParticipants in this arm will take extended-release sodium oxybate
Non-extended-release oxybatesACTIVE_COMPARATORParticipants in this arm will take non-extended-release sodium oxybate

Interventions

NameTypeDescription
Extended-release sodium oxybateDRUGParticipants 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 oxybatesDRUGParticipants 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)
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Eligibility Criteria

Age Range7 Years to 17 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: 1. Participants must be under the care of a doctor at the Stanford Sleep Clinic. 2. 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 ...

Countries:United States
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Frequently asked questions about oxybate

What is oxybate used for?

Oxybate, also known as extended-release sodium oxybate, is being investigated for the treatment of Narcolepsy Type 1 (NT1) in children. It is a small molecule in the neurology therapeutic area. The drug is currently in Phase 2 clinical development and is not yet approved for this use.

What does oxybate target?

Oxybate is a small molecule being studied for Narcolepsy Type 1 (NT1). The specific molecular target or target class has not been disclosed in the available information. The drug is being evaluated for its effects on symptoms of NT1 in a pediatric population.

Who makes oxybate?

Oxybate is being developed by Avadel Pharmaceuticals plc, a biopharmaceutical company listed on the stock exchange under the ticker symbol AVDL. The company is conducting clinical trials to evaluate the safety and efficacy of extended-release sodium oxybate in children with Narcolepsy Type 1.

What phase is oxybate in?

Oxybate is currently in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 2 trial is actively recruiting participants to study the extended-release formulation of sodium oxybate in children with Narcolepsy Type 1.

What clinical trials is oxybate in?

Oxybate is being studied in a Phase 2 clinical trial with the identifier NCT06809803, titled 'Extended-release Sodium Oxybate in Children'. This randomized, active-controlled study is recruiting 36 participants aged 7 years and older with Narcolepsy Type 1 in the United States. The trial is not double-blinded and is biomarker-selected.

Is oxybate the same as extended-release sodium oxybate?

Yes, oxybate is also known as extended-release sodium oxybate. This alternative name is used in clinical trial documentation and refers to the same drug product being developed by Avadel Pharmaceuticals for the treatment of Narcolepsy Type 1 in children.