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

Single Center, Therapeutic Exploratory Clinical Trial to Evaluate the Safety of Sodium Oxybate (Xyrem) 500 mg/mL Oral Solution on Potential Endocrine Changes at Currently Labeled Therapeutic Dose Regimens (4.5 - 9 g/Day Divided Into Two Equal Doses) During 12 Weeks of Treatment of Cataplexy in Adult Patients With Narcolepsy.

Status

Completed

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00345800

Enrollment

Registered

2006-06-29

Start date

2006-04-10

Completion date

2008-01-22

Last updated

2022-04-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Narcolepsy With Cataplexy

Keywords

Endocrine Evaluation cataplexy, sodium oxybate (xyrem)

Brief summary

To monitor for endocrine changes in response to treatment of cataplexy with Xyrem, to focus on the hypothalamic pituitary axis and to confirm the safety of Xyrem on potential endocrine changes.

Interventions

DRUGSodium Oxybate (Xyrem)

* 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

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion criteria

Inclusion Criteria: * Narcoleptic patients with cataplexy

Exclusion criteria

* Subjects not diagnosed with narcolepsy with cataplexy

Design outcomes

Primary

Measure	Time frame	Description
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of IGF-1 was done from blood sampled about 10 hours after bedtime on Visit 2.
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions After 1 Month of Treatment (Visit 3)	After 1 month of treatment (Visit 3)	An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 3.
The Insulin-like Growth Factor 1 (IGF-1) Measured in Fasting Conditions After 3 Months of Treatment (Visit 4)	After 3 months of treatment (Visit 4)	An assay of IGF-1 was done from blood sampled about 10 hours postdose on Visit 4.

Secondary

Measure	Time frame	Description
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3	Visit 3 (approximately 1 month)	Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying GH.
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4	Visit 4 (approximately 3 months)	Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying GH.
Cortisol Measured at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	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.
Cortisol Measured at Visit 3	Visit 3 (approximately 1 month)	Blood was sampled predose and and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 3 for assaying cortisol.
Cortisol Measured at Visit 4	Visit 4 (approximately 3 months)	Blood was sampled predose and 1, 2, 4, 8, 12, 16, and 20 hours postdose at Visit 4 for assaying cortisol.
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of ACTH was done from blood sampled about 10 hours after bedtime on Visit 2.
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 3.
The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of ACTH was done from blood sampled about 10 hours postdose on Visit 4.
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of DHEA-S was done from blood sampled about 10 hours after bedtime on Visit 2.
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 3.
The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of DHEA-S was done from blood sampled about 10 hours postdose on Visit 4.
The Prolactin Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of prolactin was done from blood sampled about 10 hours after bedtime on Visit 2.
The Prolactin Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 3.
The Prolactin Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of prolactin was done from blood sampled about 10 hours postdose on Visit 4.
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of TSH was done from blood sampled about 10 hours after bedtime on Visit 2.
The Total Thyroxin (T4) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of T4 was done from blood sampled about 10 hours postdose on Visit 3.
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of TSH was done from blood sampled about 10 hours postdose on Visit 4.
The Total Thyroxin (T4) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of T4 was done from blood sampled about 10 hours after bedtime on Visit 2.
The Osmolality Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of osmolality was done from blood sampled about 10 hours after bedtime on Visit 2.
The Osmolality Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 3.
The Osmolality Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of osmolality was done from blood sampled about 10 hours postdose on Visit 4.
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of electrolytes was done from blood sampled about 10 hours after bedtime on Visit 2.
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	An assay of electrolytes was done from blood sampled about 10 hours after bedtime on Visit 2.
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 3.
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 3.
Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 4.
The Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of electrolytes was done from blood sampled about 10 hours postdose on Visit 4.
The Number of Patients Reporting at Least One Adverse Event (AE) During the Course of the Study	Visit 1 through the end of the study (approximately 4 months)	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.
The Number of Patient Withdrawal Due to Adverse Events (AEs) During the Course of the Study	Visit 1 through the end of the study (approximately 4 months)	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.
The Number of Patients Reporting at Least One Serious Adverse Event (SAE) During the Course of the Study	Visit 1 through the end of the study (approximately 4 months)	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
The Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Visit 3	Visit 3 (approximately 1 month)	An assay of TSH was done from blood sampled about 10 hours postdose on Visit 3.
The Total Thyroxin (T4) Measured in Fasting Conditions at Visit 4	Visit 4 (approximately 3 months)	An assay of T4 was done from blood sampled about 10 hours postdose on Visit 4.
The Circadian Rhythm of the Growth Hormone (GH) Measured at Baseline (Visit 2)	Baseline (Visit 2) - approximately 1 day	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.

Countries

Belgium

Participant flow

Recruitment details

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

Pre-assignment details

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.

Participants by arm

Arm	Count
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	25
Total	25

Baseline characteristics

Characteristic	Sodium Oxybate
Age, Categorical <=18 years	1 Participants
Age, Categorical >=65 years	1 Participants
Age, Categorical Between 18 and 65 years	23 Participants
Age, Continuous	38.99 years STANDARD_DEVIATION 13.94
Sex: Female, Male Female	12 Participants
Sex: Female, Male Male	13 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	0 / 25
other Total, other adverse events	15 / 25
serious Total, serious adverse events	1 / 25

Outcome results

Primary