A 12-week open-label, multicenter study evaluating the safety and patient-reported efficacy of sodium oxybate in patients with narcolepsy and cataplexy
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Sleep medicine ; vol. 16, no. 1, pp. 52-58.Publisher(s)
ElsevierAuthor(s)
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Abstract(s)
Objective: This study aimed to evaluate safety and efficacy of sodium oxybate (SXB) titrated to effect.
Methods: SXB-naive patients who had participated in a randomized SXB clinical trial and had not been
titrated to adequate clinical effect were initiated on open-label SXB at 4.5 g/night and titrated in 1.5-g
increments to 6, 7.5, or 9 g/night or down to 3 g/night, based on individual clinical response. Treatment
was 12 weeks; safety was the primary outcome. Efficacy was evaluated using the Narcolepsy Symptom
Assessment Questionnaire (NSAQ), a five-point scale (“much improved” to “much worse”) that assessed
changes from baseline in specific symptoms. Response was defined as “much improved” or “somewhat
improved” overall at weeks 6 and 12.
Results: Of 202 patients, 171 (85%) completed treatment; final doses were 3 g (n = 5), 4.5 g (n = 29), 6 g
(n = 80), 7.5 g (n = 66), and 9 g (n = 22). Adverse events (AEs) were reported in 114 patients (56%), serious
AEs in five (2%). The most common AEs were nausea (10%), headache (7%), and dizziness (5%). Response
rate was 92% at week 6 and 90% at week 12; most patients reported improvements in all individual symptoms.
Overall, 60% of patients rated their symptoms at 12weeks as “much improved,” and this improvement
was dose dependent.
Conclusions: The SXB safety profile was consistent with parent trials. Ninety percent of patients
reported improvements as measured by the NSAQ.
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