Efficacy and safety of calcium, magnesium, potassium, and sodium oxybates (lower-sodium oxybate [LXB]; JZP-258) in a placebo-controlled, double-blind, randomized withdrawal study in adults with narcolepsy with cataplexy
Jazyk angličtina Země Spojené státy americké Médium print
Typ dokumentu časopisecké články, randomizované kontrolované studie, práce podpořená grantem
PubMed
33184650
PubMed Central
PMC7953213
DOI
10.1093/sleep/zsaa206
PII: 5923328
Knihovny.cz E-zdroje
- Klíčová slova
- JZP-258, cataplexy, excessive daytime sleepiness, narcolepsy, sodium oxybate,
- MeSH
- dospělí MeSH
- draslík MeSH
- dvojitá slepá metoda MeSH
- hořčík MeSH
- kataplexie * farmakoterapie MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- narkolepsie * farmakoterapie MeSH
- oxybát sodný * škodlivé účinky terapeutické užití MeSH
- senioři MeSH
- sodík MeSH
- vápník MeSH
- výsledek terapie MeSH
- Check Tag
- dospělí MeSH
- lidé středního věku MeSH
- lidé MeSH
- mladiství MeSH
- mladý dospělý MeSH
- senioři MeSH
- Publikační typ
- časopisecké články MeSH
- práce podpořená grantem MeSH
- randomizované kontrolované studie MeSH
- Názvy látek
- draslík MeSH
- hořčík MeSH
- oxybát sodný * MeSH
- sodík MeSH
- vápník MeSH
STUDY OBJECTIVES: Evaluate efficacy and safety of lower-sodium oxybate (LXB), a novel oxybate medication with 92% less sodium than sodium oxybate (SXB). METHODS: Adults aged 18-70 years with narcolepsy with cataplexy were eligible. The study included a ≤30-day screening period; a 12-week, open-label, optimized treatment and titration period to transition to LXB from previous medications for the treatment of cataplexy; a 2-week stable-dose period (SDP); a 2-week, double-blind, randomized withdrawal period (DBRWP); and a 2-week safety follow-up. During DBRWP, participants were randomized 1:1 to placebo or to continue LXB treatment. RESULTS: Efficacy was assessed in 134 participants who received randomized treatment, and safety was assessed in all enrolled participants (N = 201). Statistically significant worsening of symptoms was observed in participants randomized to placebo, with median (first quartile [Q1], third quartile [Q3]) change in weekly number of cataplexy attacks from SDP to DBRWP (primary efficacy endpoint) in the placebo group of 2.35 (0.00, 11.61) versus 0.00 (-0.49, 1.75) in the LXB group (p < 0.0001; mean [standard deviation, SD] change: 11.46 [24.751] vs 0.12 [5.772]), and median (Q1, Q3) change in Epworth Sleepiness Scale score (key secondary efficacy endpoint) of 2.0 (0.0, 5.0) in the placebo group versus 0.0 (-1.0, 1.0) in the LXB group (p < 0.0001; mean [SD] change: 3.0 [4.68] vs 0.0 [2.90]). The most common treatment-emergent adverse events with LXB were headache (20.4%), nausea (12.9%), and dizziness (10.4%). CONCLUSIONS: Efficacy of LXB for the treatment of cataplexy and excessive daytime sleepiness was demonstrated. The safety profile of LXB was consistent with SXB. CLINICAL TRIAL REGISTRATION: NCT03030599.
Biostatistics Jazz Pharmaceuticals Inc Palo Alto CA
Department of Neurology Albert Einstein College of Medicine Bronx NY
Department of Neurology University of Montpellier INSERM U1061 Montpellier France
Global Clinical Development Jazz Pharmaceuticals Inc Palo Alto CA
Helsinki Sleep Clinic Vitalmed Research Center Helsinki Finland
Neurophysiology and Sleep Disorders Unit Hospital Vithas Nuestra Señora de America Madrid Spain
School of Medicine University of South Carolina Columbia SC
Sleep and Wake Disorders Centre Department of Neurology Gui de Chauliac Hospital Montpellier France
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ClinicalTrials.gov
NCT03030599