Brief Summary
The purpose of this study is to determine whether once-nightly FT218 is safe and effective for the treatment of excessive daytime sleepiness and cataplexy in subjects with narcolepsy.
Brief Title
Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy
Categories
Completion Date
Completion Date Type
Actual
Conditions
Excessive Daytime Sleepiness
Cataplexy
Narcolepsy
Eligibility Criteria
Inclusion Criteria:
1. Male or female subjects 16 years of age or older
2. Willing and able to give written informed consent for study participation. For young adults (16 and 17 years old) who have not reached the age of majority they must be capable of giving assent and consent from a legally authorized guardian must be obtained, as required by local laws and regulations
3. Documented evidence of a diagnosis of NT1 (type 1 narcolepsy) or NT2 (type 2 narcolepsy) as, in part, determined by an overnight PSG (polysomnography) and next-day MSLT (maintenance of sleep latency test) with 2 or more SOREMPs (sleep onset REM) with mean sleep latency in the pathological range i.e. \< 8 minutes and meeting the NT1 and NT2 as defined by the International Classification of Sleep Disorders -3 criteria.
4. Current continuing presence of EDS (excessive daytime sleepiness) as defined by subject report for the last 3 months and an ESS (Epworth sleepiness scale) \> 10
5. For NT1 only, current continuing presence of cataplexy as defined by subject report for the last 3 months
6. Subjects may use concomitant stimulants, but must comply with the following:
1. They must be on a stable dose of stimulants for at least 3 weeks prior to starting the screening process for this study; AND
2. They must use the same stimulant regimen throughout the entire study period, including during screening and posttreatment periods
3. They must discontinue all anti cataplexy drugs
7. Addition inclusion criteria per protocol
Exclusion criteria
1. Any prior use of sodium oxybate is allowed in the study but within the following exclusions:
1. Previous dosing must have been limited to no more than 4.5g per night
2. Patient should not have taken sodium oxybate for more than 2 weeks.
3. All previous dosing must not have occurred within the last year prior to entry to the study.
2. Current use of sodium valproate
3. Any use of the following prohibited medications for the duration of the clinical study:
1. Anticonvulsants
2. Clonidine
3. SSRIs (selective serotonin re-uptake inhibitors) and serotonin and norepinephrine re-uptake inhibitors (SNRIs)
4. MAOIs (monoamine oxidase inhibitors)
5. TCAs (tricyclic antidepressants)
6. Hypnotics
7. Anxiolytics
8. Sedating antihistamines
9. Antipsychotics
10. Other experimental medications designed to treat narcolepsy, cataplexy or any other condition
4. Treatment with any investigational products within 3 months before study enrollment
5. Any drug known to affect sleep-wake function. Concomitant stimulant use is permitted
6. Additional exclusion criteria per protocol
1. Male or female subjects 16 years of age or older
2. Willing and able to give written informed consent for study participation. For young adults (16 and 17 years old) who have not reached the age of majority they must be capable of giving assent and consent from a legally authorized guardian must be obtained, as required by local laws and regulations
3. Documented evidence of a diagnosis of NT1 (type 1 narcolepsy) or NT2 (type 2 narcolepsy) as, in part, determined by an overnight PSG (polysomnography) and next-day MSLT (maintenance of sleep latency test) with 2 or more SOREMPs (sleep onset REM) with mean sleep latency in the pathological range i.e. \< 8 minutes and meeting the NT1 and NT2 as defined by the International Classification of Sleep Disorders -3 criteria.
4. Current continuing presence of EDS (excessive daytime sleepiness) as defined by subject report for the last 3 months and an ESS (Epworth sleepiness scale) \> 10
5. For NT1 only, current continuing presence of cataplexy as defined by subject report for the last 3 months
6. Subjects may use concomitant stimulants, but must comply with the following:
1. They must be on a stable dose of stimulants for at least 3 weeks prior to starting the screening process for this study; AND
2. They must use the same stimulant regimen throughout the entire study period, including during screening and posttreatment periods
3. They must discontinue all anti cataplexy drugs
7. Addition inclusion criteria per protocol
Exclusion criteria
1. Any prior use of sodium oxybate is allowed in the study but within the following exclusions:
1. Previous dosing must have been limited to no more than 4.5g per night
2. Patient should not have taken sodium oxybate for more than 2 weeks.
3. All previous dosing must not have occurred within the last year prior to entry to the study.
2. Current use of sodium valproate
3. Any use of the following prohibited medications for the duration of the clinical study:
1. Anticonvulsants
2. Clonidine
3. SSRIs (selective serotonin re-uptake inhibitors) and serotonin and norepinephrine re-uptake inhibitors (SNRIs)
4. MAOIs (monoamine oxidase inhibitors)
5. TCAs (tricyclic antidepressants)
6. Hypnotics
7. Anxiolytics
8. Sedating antihistamines
9. Antipsychotics
10. Other experimental medications designed to treat narcolepsy, cataplexy or any other condition
4. Treatment with any investigational products within 3 months before study enrollment
5. Any drug known to affect sleep-wake function. Concomitant stimulant use is permitted
6. Additional exclusion criteria per protocol
Inclusion Criteria
Inclusion Criteria:
1. Male or female subjects 16 years of age or older
2. Willing and able to give written informed consent for study participation. For young adults (16 and 17 years old) who have not reached the age of majority they must be capable of giving assent and consent from a legally authorized guardian must be obtained, as required by local laws and regulations
3. Documented evidence of a diagnosis of NT1 (type 1 narcolepsy) or NT2 (type 2 narcolepsy) as, in part, determined by an overnight PSG (polysomnography) and next-day MSLT (maintenance of sleep latency test) with 2 or more SOREMPs (sleep onset REM) with mean sleep latency in the pathological range i.e. \< 8 minutes and meeting the NT1 and NT2 as defined by the International Classification of Sleep Disorders -3 criteria.
4. Current continuing presence of EDS (excessive daytime sleepiness) as defined by subject report for the last 3 months and an ESS (Epworth sleepiness scale) \> 10
5. For NT1 only, current continuing presence of cataplexy as defined by subject report for the last 3 months
6. Subjects may use concomitant stimulants, but must comply with the following:
1. They must be on a stable dose of stimulants for at least 3 weeks prior to starting the screening process for this study; AND
2. They must use the same stimulant regimen throughout the entire study period, including during screening and posttreatment periods
3. They must discontinue all anti cataplexy drugs
7. Addition inclusion criteria per protocol
1. Male or female subjects 16 years of age or older
2. Willing and able to give written informed consent for study participation. For young adults (16 and 17 years old) who have not reached the age of majority they must be capable of giving assent and consent from a legally authorized guardian must be obtained, as required by local laws and regulations
3. Documented evidence of a diagnosis of NT1 (type 1 narcolepsy) or NT2 (type 2 narcolepsy) as, in part, determined by an overnight PSG (polysomnography) and next-day MSLT (maintenance of sleep latency test) with 2 or more SOREMPs (sleep onset REM) with mean sleep latency in the pathological range i.e. \< 8 minutes and meeting the NT1 and NT2 as defined by the International Classification of Sleep Disorders -3 criteria.
4. Current continuing presence of EDS (excessive daytime sleepiness) as defined by subject report for the last 3 months and an ESS (Epworth sleepiness scale) \> 10
5. For NT1 only, current continuing presence of cataplexy as defined by subject report for the last 3 months
6. Subjects may use concomitant stimulants, but must comply with the following:
1. They must be on a stable dose of stimulants for at least 3 weeks prior to starting the screening process for this study; AND
2. They must use the same stimulant regimen throughout the entire study period, including during screening and posttreatment periods
3. They must discontinue all anti cataplexy drugs
7. Addition inclusion criteria per protocol
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
16 Years
NCT Id
NCT02720744
Org Class
Industry
Org Full Name
Avadel
Org Study Id
CLFT218-1501
Overall Status
Completed
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Double-blind, Randomized, Placebo Controlled, Two Arm Multi-center Study to Assess the Efficacy and Safety of a Once Nightly Formulation of Sodium Oxybate for Extended-Release Oral Suspension (FT218) for the Treatment of Excessive Daytime Sleepiness and Cataplexy in Subjects With Narcolepsy
Primary Outcomes
Outcome Description
Change from Baseline for MWT, which is the mean latency across 5 naps, averaged over the test day
Outcome Measure
Maintenance of Wakefulness Test (MWT)
Outcome Time Frame
Study Visit 8 at 14 weeks
Outcome Description
The CGI is the clinician's global impression of improvement in daytime sleepiness. For the CGI, a GLIMMIX (generalized linear mixed models) model for binomial data with logit link was used to analyze the categorized CGI response, i.e., the proportions of subjects who were Very Much Improved or Much Improved as compared to Screening
Outcome Measure
Proportion of Patients That Were Very Much Improved or Much Improved on Clinical Global Impression of Improvement as Compared to Screening
Outcome Time Frame
Study Visit 8 at 14 weeks
Outcome Description
Mean number of cataplexy events recorded on the Sleep and Symptom Daily Diary during the period
Outcome Measure
Number of Cataplexy Attacks at Visit 8 (Week 14) as Compared to Baseline
Outcome Time Frame
Visit 8 - Change from Baseline at 14 Weeks
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
16
Investigators
Investigator Type
Principal Investigator
Investigator Name
Michael Thorpy
Investigator Email
michael.thorpy@einsteinmed.org
Investigator Phone
718-920-4841