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

Study Overview
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Brief Summary
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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.

Official Title
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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.

Conditions
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Narcolepsy With Cataplexy

Intervention / Treatment
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* Drug: Sodium Oxybate (Xyrem)


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Inclusion Criteria: Narcoleptic patients with cataplexy Exclusion Criteria: Subjects not diagnosed with narcolepsy with cataplexy Exclusion Criteria:

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Sodium Oxybate<br>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)<br>* 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<br>* Other names: Xyrem;|

What is the study measuring?
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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) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Endocrine Evaluation cataplexy, sodium oxybate (xyrem)