Trial Outcomes & Findings for Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Disturbance in Parkinson's Disease (PD) (NCT NCT00641186)

Last Updated: 2022-10-06

Results Overview

Sleep and Fatigue was measured using the following scales before and after sodium oxybate therapy Scales used to measure sleep and fatigue: Epworth Sleepiness Scale (ESS) where score ranges from 0-24 where 0 = no sleep problems and 24 = excessive sleep problems that should seek medical attention; Fatigue Severity Scale (FSS) where the score ranges from 9-36 where 9 = no fatigue and 36 = severe fatigue; Pittsburgh Sleep Quality Inventory (PSQI) where the score ranges from 0-21 where any score greater than 5 indicates a great sleep disturbance; 36-Item Short Form Health Survey (SF-36) where the score ranges 0-100 where 0 = the worst health status and 100 = the best health status. \* Findings are reported for 28 subjects. The Full Range values reported reflect the measured minimum and maximum values.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

30 participants

Primary outcome timeframe

8 weeks

Results posted on

2022-10-06

Participant Flow

Participant milestones

Participant milestones
Measure	Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Dist Xyrem arm, single arm study for excessive daytime sleepiness and sleep disturbance in PD
Overall Study STARTED	30
Overall Study COMPLETED	27
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Dist Xyrem arm, single arm study for excessive daytime sleepiness and sleep disturbance in PD
Overall Study Adverse Event	3

Baseline Characteristics

Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Disturbance in Parkinson's Disease (PD)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment Group n=30 Participants Active treatment group with Xyrem in PD
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=99 Participants
Age, Categorical >=65 years	10 Participants n=99 Participants
Sex: Female, Male Female	6 Participants n=99 Participants
Sex: Female, Male Male	24 Participants n=99 Participants
Region of Enrollment United States	30 participants n=99 Participants

PRIMARY outcome

Timeframe: 8 weeks

Outcome measures

Outcome measures
Measure	Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Disturbance n=28 Participants Xyrem arm, single arm study for excessive sleep disturbance in PD
Sleep and Fatigue Before Sodium Oxybate Therapy (baseline) ESS	15.6 units on a scale Interval 0.0 to 24.0
Sleep and Fatigue After Sodium Oxybate Therapy (8 weeks) ESS	9.0 units on a scale Interval 0.0 to 24.0
Sleep and Fatigue Before Sodium Oxybate Therapy (baseline) PSQI	10.9 units on a scale Interval 0.0 to 21.0
Sleep and Fatigue After Sodium Oxybate Therapy (8 weeks) PSQI	6.6 units on a scale Interval 0.0 to 21.0
Sleep and Fatigue Before Sodium Oxybate Therapy (baseline) FSS	32.9 units on a scale Interval 9.0 to 36.0
Sleep and Fatigue After Sodium Oxybate Therapy (8 weeks) FSS	26.3 units on a scale Interval 9.0 to 36.0
Sleep and Fatigue Before Sodium Oxybate Therapy (baseline) SF-36	95.7 units on a scale Interval 0.0 to 100.0
Sleep and Fatigue After Sodium Oxybate Therapy (8 weeks) SF-36	92.3 units on a scale Interval 0.0 to 100.0

SECONDARY outcome

Timeframe: 8 weeks

Population: A polysomnography was done before and after therapy as a secondary measure. The results are below.

Polysomnography, also called a sleep study, is a comprehensive test used to diagnose sleep disorders. The minimum score would be 0, the maximum score would be 30. A score of 0 - 4 = normal sleep, a score of 5 - 14 = a mild level of sleep disturbance, a score of 15 - 30 is moderate level of sleep disturbance and a score of 30 is severe sleep disturbance.

Outcome measures

Outcome measures
Measure	Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Disturbance n=27 Participants Xyrem arm, single arm study for excessive sleep disturbance in PD
Polysomnography (PSG) Mean Polysomnography Before Therapy	7 units on a scale Interval 0.0 to 14.0
Polysomnography (PSG) Mean Polysomnography After Therapy	13 units on a scale Interval 0.0 to 30.0

Adverse Events

All Subjects

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	All Subjects n=30 participants at risk Adverse events probably or definitely related to the drug included: nocturia/enuresis (n=3), dizziness and nausea (n=1), dizziness with daytime sleepiness reduced alertness (n = 1), dizziness with rebound morning tremor (n=1). Additional adverse events that were considered not related to the study drug included: constipation (n=1),1 delusions (n=1), and, in a single subject, bradycardia, anxiety, depression, and edema. Twenty-one of 30 subjects (70%) reported no adverse events.
Renal and urinary disorders enuresis	10.0% 3/30 • Number of events 3 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Nervous system disorders rebounding morning tremor	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
General disorders dizziness	10.0% 3/30 • Number of events 3 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
General disorders daytime sleepiness	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Gastrointestinal disorders constipation	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Psychiatric disorders delusions	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
General disorders bradycardia	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Psychiatric disorders Anxiety	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Psychiatric disorders depression	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
Blood and lymphatic system disorders edema	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.
General disorders nausea	3.3% 1/30 • Number of events 1 • Screening through follow-up for a total duration 56 days. Any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with that treatment.

Additional Information

Christine Hunter

Baylor College of Medicine

Phone: 713-798-3951

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place