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oxybate

Phase 2

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

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedACTIVE_CONTROLLEDBiomarker
Total Trials1
Total Enrollment36
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06809803Extended-release Sodium Oxybate in ChildrenPHASE2 RECRUITING 36Oct 27, 2025Jul 1, 2027Nov 6, 20251 United States
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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 — 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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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT06809803studyFirstPostDate: changed