Trial Outcomes & Findings for Exploratory Clinical Study to Evaluate Sodium Oxybate (Xyrem) on Potential Endocrine Changes (NCT NCT00345800)

NCT ID: NCT00345800

Last Updated: 2022-04-08

Results Overview

An assay of IGF-1 was done from blood sampled about 10 hours after bedtime on Visit 2.

• Recruitment status: COMPLETED
• Study phase: PHASE1
• Target enrollment: 25 participants
• Primary outcome timeframe: Baseline (Visit 2) - approximately 1 day
• Results posted on: 2022-04-08

Participant Flow

This single-center, therapeutic, exploratory study started to enroll subjects in April 2006.

Participant Flow refers to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Participant milestones

Measure	Sodium Oxybate Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Overall Study STARTED	25
Overall Study COMPLETED	25
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Exploratory Clinical Study to Evaluate Sodium Oxybate (Xyrem) on Potential Endocrine Changes

Baseline characteristics by cohort

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	23 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	38.99 years STANDARD_DEVIATION 13.94 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of IGF-1 was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions at Baseline (Visit 2)	207.4 ng/mL Standard Deviation 86.5

PRIMARY outcome

Timeframe: After 1 month of treatment (Visit 3)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions After 1 Month of Treatment (Visit 3)	200.4 ng/mL Standard Deviation 77.7

PRIMARY outcome

Timeframe: After 3 months of treatment (Visit 4)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions After 3 Months of Treatment (Visit 4)	210.2 ng/mL Standard Deviation 90.3

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled at Baseline (Visit 2) at bedtime 10:00 pm and 1, 2, 4, 8, 12, 16, and 20 hours after bedtime for assaying GH.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) Bed time	1.821 ng/mL Standard Deviation 4.844
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 1h	1.225 ng/mL Standard Deviation 2.895
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 2h	1.408 ng/mL Standard Deviation 1.656
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 4h	1.236 ng/mL Standard Deviation 1.904
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 8h	0.576 ng/mL Standard Deviation 0.632
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 12h	0.293 ng/mL Standard Deviation 0.310
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 16h	0.309 ng/mL Standard Deviation 0.282
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2) 20h	0.313 ng/mL Standard Deviation 0.362

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying GH.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 Pre-dose	2.432 ng/mL Standard Deviation 7.118
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 1h	5.914 ng/mL Standard Deviation 9.743
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 2h	5.238 ng/mL Standard Deviation 8.557
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 4h	3.018 ng/mL Standard Deviation 4.332
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 8h	0.256 ng/mL Standard Deviation 0.326
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 12h	0.308 ng/mL Standard Deviation 0.664
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 16h	0.460 ng/mL Standard Deviation 0.767
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3 20h	0.306 ng/mL Standard Deviation 0.342

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying GH.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 Pre-dose	0.816 ng/mL Standard Deviation 1.372
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 1h	3.804 ng/mL Standard Deviation 3.173
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 2h	3.864 ng/mL Standard Deviation 5.731
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 4h	2.712 ng/mL Standard Deviation 3.178
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 8h	0.224 ng/mL Standard Deviation 0.292
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 12h	0.420 ng/mL Standard Deviation 1.355
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 16h	0.296 ng/mL Standard Deviation 0.491
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4 20h	0.244 ng/mL Standard Deviation 0.390

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled at Baseline (Visit 2) at bedtime 10:00 pm and 1, 2, 4, 8, 12, 16, and 20 hours after bedtime for assaying cortisol.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Cortisol Measured at Baseline (Visit 2) Bed time	42.21 ug/L Standard Deviation 28.53
Cortisol Measured at Baseline (Visit 2) 1h	27.91 ug/L Standard Deviation 17.09
Cortisol Measured at Baseline (Visit 2) 2h	22.80 ug/L Standard Deviation 14.54
Cortisol Measured at Baseline (Visit 2) 4h	36.06 ug/L Standard Deviation 28.46
Cortisol Measured at Baseline (Visit 2) 8h	115.79 ug/L Standard Deviation 52.33
Cortisol Measured at Baseline (Visit 2) 12h	143.39 ug/L Standard Deviation 70.67
Cortisol Measured at Baseline (Visit 2) 16h	90.84 ug/L Standard Deviation 34.83
Cortisol Measured at Baseline (Visit 2) 20h	68.48 ug/L Standard Deviation 40.77

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled predose and and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying cortisol.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Cortisol Measured at Visit 3 Pre-dose	35.84 ug/L Standard Deviation 27.83
Cortisol Measured at Visit 3 1h	24.75 ug/L Standard Deviation 18.20
Cortisol Measured at Visit 3 2h	34.62 ug/L Standard Deviation 31.93
Cortisol Measured at Visit 3 4h	44.28 ug/L Standard Deviation 36.64
Cortisol Measured at Visit 3 8h	95.61 ug/L Standard Deviation 57.14
Cortisol Measured at Visit 3 12h	130.21 ug/L Standard Deviation 61.28
Cortisol Measured at Visit 3 16h	77.33 ug/L Standard Deviation 32.93
Cortisol Measured at Visit 3 20h	70.49 ug/L Standard Deviation 33.71

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying cortisol.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Cortisol Measured at Visit 4 1h	25.61 ug/L Standard Deviation 19.06
Cortisol Measured at Visit 4 Pre-dose	34.46 ug/L Standard Deviation 24.85
Cortisol Measured at Visit 4 2h	30.06 ug/L Standard Deviation 24.40
Cortisol Measured at Visit 4 4h	45.27 ug/L Standard Deviation 35.65
Cortisol Measured at Visit 4 8h	113.84 ug/L Standard Deviation 71.18
Cortisol Measured at Visit 4 12h	146.33 ug/L Standard Deviation 64.42
Cortisol Measured at Visit 4 16h	92.48 ug/L Standard Deviation 30.94
Cortisol Measured at Visit 4 20h	75.55 ug/L Standard Deviation 38.01

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of ACTH was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Baseline (Visit 2)	65.6 pg/mL Standard Deviation 46.7

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Visit 3	62.8 pg/mL Standard Deviation 37.9

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Visit 4	54.8 pg/mL Standard Deviation 27.3

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of DHEA-S was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Baseline (Visit 2)	1988.2 ug/L Standard Deviation 1283.3

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Visit 3	2200.9 ug/L Standard Deviation 1517.5

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Visit 4	2130.7 ug/L Standard Deviation 1450.4

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of prolactin was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Prolactin Measured in Fasting Conditions at Baseline (Visit 2)	356.8 mUI/L Standard Deviation 185.9

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Prolactin Measured in Fasting Conditions at Visit 3	280.0 mUI/L Standard Deviation 162.8

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Prolactin Measured in Fasting Conditions at Visit 4	258.4 mUI/L Standard Deviation 113.6

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of TSH was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Baseline (Visit 2)	1.366 uU/mL Standard Deviation 0.622

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of TSH was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Visit 3	1.292 uU/mL Standard Deviation 0.599

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of TSH was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Visit 4	1.425 uU/mL Standard Deviation 0.818

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of T4 was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Total Thyroxin (T4) Measured in Fasting Conditions at Baseline (Visit 2)	11.34 pg/mL Standard Deviation 1.68

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of T4 was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Total Thyroxin (T4) Measured in Fasting Conditions at Visit 3	11.47 pg/mL Standard Deviation 2.10

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of T4 was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Total Thyroxin (T4) Measured in Fasting Conditions at Visit 4	11.65 pg/mL Standard Deviation 1.78

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of osmolality was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Osmolality Measured in Fasting Conditions at Baseline (Visit 2)	291.6 mosm/kg Standard Deviation 4.5

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Osmolality Measured in Fasting Conditions at Visit 3	292.2 mosm/kg Standard Deviation 4.9

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Osmolality Measured in Fasting Conditions at Visit 4	292.1 mosm/kg Standard Deviation 5.5

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Baseline (Visit 2) Na (Sodium in mmol/L)	141.4 mmol/L Standard Deviation 1.9
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Baseline (Visit 2) K (Potassium in mmol/L)	3.88 mmol/L Standard Deviation 0.21
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Baseline (Visit 2) Ca (Calcium in mmol/L)	2.257 mmol/L Standard Deviation 0.084

SECONDARY outcome

Timeframe: Baseline (Visit 2) - approximately 1 day

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours after bedtime on Visit 2.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Baseline (Visit 2)	34.4 mg/L Standard Deviation 6.0

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 3 Na (Sodium in mmol/L)	141.9 mmol/L Standard Deviation 2.1
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 3 K (Potassium in mmol/L)	3.90 mmol/L Standard Deviation 0.25
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 3 Ca (Calcium in mmol/L)	2.334 mmol/L Standard Deviation 0.071

SECONDARY outcome

Timeframe: Visit 3 (approximately 1 month)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 3.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 3	34.2 mg/L Standard Deviation 4.5

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 4 Na (Sodium in mmol/L)	141.2 mmol/L Standard Deviation 1.9
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 4 K (Potassium in mmol/L)	3.80 mmol/L Standard Deviation 0.27
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 4 Ca (Calcium in mmol/L)	2.290 mmol/L Standard Deviation 0.063

SECONDARY outcome

Timeframe: Visit 4 (approximately 3 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 4.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 4	35.0 mg/L Standard Deviation 3.5

SECONDARY outcome

Timeframe: Visit 1 through the end of the study (approximately 4 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An AE was classified as a treatment-emergent AE (TEAE) if its onset date and time was on or after the first study drug administration. Number of subjects with at least one TEAE is reported below.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Number of Patients Reporting at Least One Adverse Event (AE) During the Course of the Study	20 Participants

SECONDARY outcome

Timeframe: Visit 1 through the end of the study (approximately 4 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

An AE was classified as a treatment-emergent AE (TEAE) if its onset date and time was on or after the first study drug administration. Number of subjects with TEAE that led to temporarily discontinuation of study drug is reported below.

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Number of Patient Withdrawal Due to Adverse Events (AEs) During the Course of the Study	1 Participants

SECONDARY outcome

Timeframe: Visit 1 through the end of the study (approximately 4 months)

Population: Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

A Serious Adverse Event is any untoward medical occurrence that at any dose • results in death, • is life threatening, • requires in-patient hospitalization or prolongation of existing hospitalization, • results in persistent or significant disability/incapacity • is a congenital anomaly/birth defect

Outcome measures

Measure	Sodium Oxybate n=25 Participants Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
The Number of Patients Reporting at Least One Serious Adverse Event (SAE) During the Course of the Study	1 Participants

Adverse Events

Sodium Oxybate

Serious events: 1 serious events

Other events: 15 other events

Deaths: 0 deaths

Serious adverse events

Measure	Sodium Oxybate n=25 participants at risk Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Gastrointestinal disorders Rectal haemorrhage	4.0% 1/25 • Number of events 1 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Other adverse events

Measure	Sodium Oxybate n=25 participants at risk Active Substance: Sodium Oxybate Pharmaceutical form: Oral Solution Concentration: 500 mg/mL oral solution from 4.5 to 9 g/day divided into two equal doses during 12 weeks Route of administration: Oral
Gastrointestinal disorders Nausea	20.0% 5/25 • Number of events 7 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Gastrointestinal disorders Abdominal pain upper	12.0% 3/25 • Number of events 7 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Gastrointestinal disorders Diarrhoea	8.0% 2/25 • Number of events 2 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Musculoskeletal and connective tissue disorders Arthralgia	8.0% 2/25 • Number of events 2 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Musculoskeletal and connective tissue disorders Osteoarthritis	8.0% 2/25 • Number of events 4 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Nervous system disorders Headache	24.0% 6/25 • Number of events 8 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Nervous system disorders Dizziness	8.0% 2/25 • Number of events 2 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Psychiatric disorders Anxiety	8.0% 2/25 • Number of events 6 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Respiratory, thoracic and mediastinal disorders Cough	8.0% 2/25 • Number of events 3 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.
Skin and subcutaneous tissue disorders Hyperhidrosis	8.0% 2/25 • Number of events 2 • Adverse Events were collected from Visit 1 through the end of the study (approximately 4 months). Adverse Events refer to the Safety Population which is identical to the Intention-to-Treat (ITT) population consisting of the 25 subjects enrolled in the study who took at least 1 dose of study medication.

Additional Information

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Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: GT60