Clinical trial NCT04006925: Treatment of REM Sleep Behavior Disorder (RBD) With Sodium Oxybate
Official title: Sodium Oxybate in Treatment-Resistant REM Sleep Behavior Disorder (RBD): A Randomized Placebo-Controlled Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: REM Sleep Behavior Disorder
Parkinson Disease
Keywords: sodium oxybate
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Jazz Pharmaceuticals
First posted: 2019-07-05
Enrollment: 24
Sex: ALL
Minimum age (years): 40
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomized, double-blind, placebo-controlled trial
Primary purpose: TREATMENT

Brief summary: This study is the first clinical trial using sodium oxybate for the treatment of REM sleep behavior disorder (RBD). Sodium oxybate is a drug approved by FDA for the treatment of narcolepsy which has been used "off label" to treat patients with severe RBD. This drug has shown to be effective and well tolerated in patients with RBD (Shneerson, 2009; Liebenthal, 2016; Moghadam, 2017).

Detailed description: Rapid eye movement sleep behavior disorder (RBD) is a condition resulting in violent dream-enactment during sleep which affects millions of individuals in the United States, however therapies for RBD are limited and cause significant side effects. As a result, despite using a combination of drugs, a large number of patients with RBD continue to act out violent dreams causing severe self-injuries or injuries to their bed partners. Prior studies and our experience have shown that sodium oxybate can be effective in these cases of treatment-resistant RBD. This study would therefore evaluate the efficacy and tolerance of sodium oxybate in this patient population.

This study is an 8-week trial comparing sodium oxybate versus placebo randomly assigned to patients with treatment-resistant RBD, i.e. individuals who have insufficiently responded or tolerated melatonin and clonazepam. The study uses a double-blind design (participants, staff, and investigators will not know which drug between active drug and placebo is given to participants), and will measure treatment efficacy based on patients, partners and clinicians report, and objective outcomes based on in-home actigraphy and in-lab polysomnography before and after intervention.

Eligibility criteria: Inclusion Criteria:

* 40-85 years old
* With or without Parkinson's disease
* Experiencing RBD episodes on average at least 2x/week or 8x/month
* Able to report RBD episodes themselves or via a partner witness

Exclusion Criteria:

* History of falls during ambulation in the last 6 months despite adequate neurologic treatment
* Requirement of an ambulatory device at home
* Inadequately treated symptomatic orthostatic hypotension
* BMI \> 35
* Untreated or uncontrolled OSA (4%AHI\>15)
* Cognitive impairment resulting in inability to comply with treatment instructions
* Pregnancy

Primary outcomes:
measure: Number of RBD Episodes in One Month (Per Patient RBD Log)
timeFrame: Assessed for 28 days at baseline and during last month of treatment (total treatment period of up to 12 weeks)
measure: Number of Severe of RBD Episodes in One Month (Per Patient RBD Log)
timeFrame: Assessed for 28 days at baseline and during last month of treatment (total treatment period of up to 12 weeks)
Secondary outcomes:
measure: Number of Responders According to the CGI Efficacy Scale (CGI-E)
timeFrame: Assessed at week 12 (end of treatment period).
measure: Number of Responders According to the CGI Improvement Scale (CGI-I)
timeFrame: Assessed at week 12 (end of treatment period).
measure: Epworth Sleepiness Scale (ESS) Score
timeFrame: Assessed at baseline and week 12
measure: RBD Episode Severity and Frequency During REM Sleep by Quantitative Video-PSG (Polysomnography) Analysis Per 10 Minutes of REM Sleep
timeFrame: Assessed at baseline and week 12 (average approximately 8 hours to assess at each time point)
measure: Change in Ambulatory Measures of Movements During Sleep Using Actigraphy
timeFrame: Assessed for 28 days at baseline and during last month of treatment (total treatment period of up to 12 weeks)
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford University
state: California
Study officials:
affiliation: Stanford University
name: Emmanuel During, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Shneerson JM. Successful treatment of REM sleep behavior disorder with sodium oxybate. Clin Neuropharmacol. 2009 May-Jun;32(3):158-9. doi: 10.1097/WNF.0b013e318193e394.
citation: Liebenthal J, Valerio J, Ruoff C, Mahowald M. A Case of Rapid Eye Movement Sleep Behavior Disorder in Parkinson Disease Treated With Sodium Oxybate. JAMA Neurol. 2016 Jan;73(1):126-7. doi: 10.1001/jamaneurol.2015.2904. No abstract available.
citation: Moghadam KK, Pizza F, Primavera A, Ferri R, Plazzi G. Sodium oxybate for idiopathic REM sleep behavior disorder: a report on two patients. Sleep Med. 2017 Apr;32:16-21. doi: 10.1016/j.sleep.2016.04.014. Epub 2016 Jun 7.