Mortimer Mamelak1, Todd Swick2, Helene Emsellem3, Jacques Montplaisir4, Chinglin Lai5, Jed Black6. 1. Department of Psychiatry, University of Toronto, Toronto, ON, Canada. Electronic address: m.mamelak@utoronto.ca. 2. Neurology and Sleep Medicine Consultants of Houston, University of Texas-Houston School of Medicine, Houston, TX, USA. 3. George Washington University School of Medicine, Washington, DC, USA; The Center for Sleep & Wake Disorders, Chevy Chase, MD, USA. 4. Department of Psychiatry, Université de Montréal, Montréal, Québec, Canada. 5. Jazz Pharmaceuticals, Inc., Palo Alto, CA, USA. 6. Jazz Pharmaceuticals, Inc., Palo Alto, CA, USA; Stanford Center for Sleep Research and Medicine, Stanford, CA, USA.
Abstract
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 12 weeks 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.
RCT Entities:
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 12 weeks 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.
Authors: Chiraag Thakrar; Kishankumar Patel; Grainne D'ancona; Brian D Kent; Alexander Nesbitt; Hugh Selsick; Joerg Steier; Ivana Rosenzweig; Adrian J Williams; Guy D Leschziner; Panagis Drakatos Journal: J Sleep Res Date: 2017-10-19 Impact factor: 3.981
Authors: Filipa Raposo Pereira; Minni T B McMaster; Yvon D A T de Vries; Nikki Polderman; Wim van den Brink; Guido A van Wingen Journal: Eur Addict Res Date: 2019-04-18 Impact factor: 3.015
Authors: Panagis Drakatos; Dimosthenis Lykouras; Grainne D'Ancona; Sean Higgins; Nadia Gildeh; Raluca Macavei; Ivana Rosenzweig; Joerg Steier; Adrian J Williams; Rexford Muza; Brian D Kent; Guy Leschziner Journal: Sleep Med Date: 2017-05-06 Impact factor: 3.492