MedPath

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

Phase 1
Completed
Conditions
Narcolepsy With Cataplexy
Interventions
Registration Number
NCT00345800
Lead Sponsor
UCB Pharma SA
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
25
Inclusion Criteria

Inclusion Criteria:

  • Narcoleptic patients with cataplexy
Exclusion Criteria
  • Subjects not diagnosed with narcolepsy with cataplexy

Exclusion Criteria:

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Sodium OxybateSodium 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
Primary Outcome Measures
NameTimeMethod
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 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 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 Outcome Measures
NameTimeMethod
The Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 3Visit 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.

Cortisol Measured at Visit 4Visit 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 Total Thyroxin (T4) Measured in Fasting Conditions at Visit 3Visit 3 (approximately 1 month)

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

The Osmolality Measured in Fasting Conditions at Visit 4Visit 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.

The Adrenocorticotropic Hormone (ACTH) Measured in Fasting Conditions at Visit 4Visit 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 Prolactin Measured in Fasting Conditions at Visit 3Visit 3 (approximately 1 month)

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

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 Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 4Visit 4 (approximately 3 months)

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

The Prolactin Measured in Fasting Conditions at Visit 4Visit 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 Visit 4Visit 4 (approximately 3 months)

An assay of TSH 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 StudyVisit 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 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.

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 3Visit 3 (approximately 1 month)

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

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 4Visit 4 (approximately 3 months)

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

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 Circadian Rhythm of the Growth Hormone (GH) Measured at Visit 4Visit 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 3Visit 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.

The Dehydroepiandrosterone Sulfate (DHEA-S) Measured in Fasting Conditions at Visit 3Visit 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 4Visit 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 Thyroid Stimulating Hormone (TSH) Measured in Fasting Conditions at Visit 3Visit 3 (approximately 1 month)

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

The Osmolality Measured in Fasting Conditions at Visit 3Visit 3 (approximately 1 month)

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

The Electrolyte Phosphate (P) Measured in Fasting Conditions at Visit 3Visit 3 (approximately 1 month)

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

The Number of Patients Reporting at Least One Serious Adverse Event (SAE) During the Course of the StudyVisit 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

Electrolytes (Na, K, Ca) Measured in Fasting Conditions at Visit 3Visit 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 4Visit 4 (approximately 3 months)

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

The Number of Patient Withdrawal Due to Adverse Events (AEs) During the Course of the StudyVisit 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.

© Copyright 2025. All Rights Reserved by MedPath