Changes in Functional Status, Work Productivity, and Daily Activities in People With Idiopathic Hypersomnia and Narcolepsy Treated With Low-Sodium Oxybate: Results From the Phase 4 DUET Study
Author(s)
Alyssa Cairns, PhD1, Logan D. Schneider, MD2, David T. Plante, MD, PhD3, Deborah A. Nichols, MS4, Teresa L. Steininger, PhD4, Douglas S. Fuller, MS5, Marisa Whalen, PharmD5, Silky Beaty, PharmD, MSPH4, Chad Ruoff, MD6.
1BioSerenity, Inc., Columbia, SC, USA, 2Stanford University Center for Sleep Sciences and Medicine, Stanford, CA, USA, 3Department of Psychiatry, University of Wisconsin–Madison, Madison, WI, USA, 4Jazz Pharmaceuticals, Palo Alto, CA, USA, 5Jazz Pharmaceuticals, Philadelphia, PA, USA, 6Mayo Clinic, Scottsdale, AZ, USA.
1BioSerenity, Inc., Columbia, SC, USA, 2Stanford University Center for Sleep Sciences and Medicine, Stanford, CA, USA, 3Department of Psychiatry, University of Wisconsin–Madison, Madison, WI, USA, 4Jazz Pharmaceuticals, Palo Alto, CA, USA, 5Jazz Pharmaceuticals, Philadelphia, PA, USA, 6Mayo Clinic, Scottsdale, AZ, USA.
Presentation Documents
OBJECTIVES: Jazz DUET (Develop hypersomnia Understanding by Evaluating low-sodium oxybate Treatment) (NCT05875974) is a phase 4, prospective, multicenter, single-arm, multiple-cohort, open-label study evaluating the effectiveness of low-sodium oxybate (LXB; calcium, magnesium, potassium, and sodium oxybates) on daytime sleepiness and functional status, including work productivity and daily activities, in people with idiopathic hypersomnia or narcolepsy (2 separate cohorts).
METHODS: DUET comprised 2- to 6-week screening (with 2-week washout for current oxybate users), 8-day baseline (BL), 2- to 8-week LXB titration, 2-week stable-dose, 8-day end of treatment (EOT), and 2-week safety follow-up periods. Exploratory outcomes included the Functional Outcomes of Sleep Questionnaire-10 (FOSQ-10) and Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP), both administered at BL and EOT.
RESULTS: Forty-six participants with idiopathic hypersomnia (80.4% female; 84.8% White; mean [SD] age, 38.1 [11.8] years) and 55 with narcolepsy (72.7% female, 80.0% White; mean [SD] age, 33.4 [12.9] years) enrolled and took ≥1 dose of LXB after BL. At study entry, 9/46 participants with idiopathic hypersomnia and 13/55 with narcolepsy were taking oxybate and washed out before BL. BL FOSQ-10 mean (SD) scores for idiopathic hypersomnia and narcolepsy, respectively, were 11.8 (2.8) and 11.4 (3.0); mean (SD) changes from BL to EOT were 3.3 (2.7) and 2.9 (2.7), respectively. BL WPAI:SHP mean (SD) values for idiopathic hypersomnia and narcolepsy, respectively, were 59.8% (19.0%) and 61.3% (23.4%) for overall work impairment and 67.5% (19.4%) and 56.2% (21.0%) for non-work-related activity impairment; mean (SD) changes from BL to EOT were −32.4% (24.6%) and −36.1% (22.2%) for overall work impairment and −37.3% (28.8%) and −23.7% (25.0%) for non-work-related activity impairment, respectively. Treatment-emergent adverse events were consistent with the known safety profile of LXB.
CONCLUSIONS: Open-label LXB was associated with improvements from BL impairment in functional status, overall work productivity, and non-work-related activities.
METHODS: DUET comprised 2- to 6-week screening (with 2-week washout for current oxybate users), 8-day baseline (BL), 2- to 8-week LXB titration, 2-week stable-dose, 8-day end of treatment (EOT), and 2-week safety follow-up periods. Exploratory outcomes included the Functional Outcomes of Sleep Questionnaire-10 (FOSQ-10) and Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI:SHP), both administered at BL and EOT.
RESULTS: Forty-six participants with idiopathic hypersomnia (80.4% female; 84.8% White; mean [SD] age, 38.1 [11.8] years) and 55 with narcolepsy (72.7% female, 80.0% White; mean [SD] age, 33.4 [12.9] years) enrolled and took ≥1 dose of LXB after BL. At study entry, 9/46 participants with idiopathic hypersomnia and 13/55 with narcolepsy were taking oxybate and washed out before BL. BL FOSQ-10 mean (SD) scores for idiopathic hypersomnia and narcolepsy, respectively, were 11.8 (2.8) and 11.4 (3.0); mean (SD) changes from BL to EOT were 3.3 (2.7) and 2.9 (2.7), respectively. BL WPAI:SHP mean (SD) values for idiopathic hypersomnia and narcolepsy, respectively, were 59.8% (19.0%) and 61.3% (23.4%) for overall work impairment and 67.5% (19.4%) and 56.2% (21.0%) for non-work-related activity impairment; mean (SD) changes from BL to EOT were −32.4% (24.6%) and −36.1% (22.2%) for overall work impairment and −37.3% (28.8%) and −23.7% (25.0%) for non-work-related activity impairment, respectively. Treatment-emergent adverse events were consistent with the known safety profile of LXB.
CONCLUSIONS: Open-label LXB was associated with improvements from BL impairment in functional status, overall work productivity, and non-work-related activities.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR3
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, SDC: Neurological Disorders