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.

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.

Study Overview

• Status: Completed
• Conditions: Narcolepsy With Cataplexy
• Intervention / Treatment: Drug: Sodium Oxybate (Xyrem)

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 1

Contacts and Locations

Study Locations

• Belgium
  • Liège, Belgium

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

Inclusion Criteria:

• Narcoleptic patients with cataplexy

Exclusion Criteria:

• Subjects not diagnosed with narcolepsy with cataplexy

Exclusion Criteria:

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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	Drug: Sodium 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; Other Names: Xyrem

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: UCB Pharma SA

Publications and helpful links

Helpful Links

• FDA Safety Alerts and Recalls
• Product Information

Study record dates

Study Major Dates

• Study Start (Actual): April 10, 2006
• Primary Completion (Actual): January 22, 2008
• Study Completion (Actual): January 22, 2008

Study Registration Dates

• First Submitted: June 28, 2006
• First Submitted That Met QC Criteria: June 28, 2006
• First Posted (Estimate): June 29, 2006

Study Record Updates

• Last Update Posted (Actual): April 8, 2022
• Last Update Submitted That Met QC Criteria: February 9, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: Endocrine Evaluation cataplexy; sodium oxybate (xyrem)
• Additional Relevant MeSH Terms: Mental Disorders; Nervous System Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Disorders of Excessive Somnolence; Narcolepsy; Cataplexy; Physiological Effects of Drugs; Central Nervous System Depressants; Anesthetics, Intravenous; Anesthetics, General; Anesthetics; Adjuvants, Anesthesia; Sodium Oxybate
• Other Study ID Numbers: C00301; 2005-004417-15 (EudraCT Number)