Trial Outcomes & Findings for Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy (NCT NCT02720744)

NCT ID: NCT02720744

Last Updated: 2022-03-22

Results Overview

Change from Baseline for MWT, which is the mean latency across 5 naps, averaged over the test day

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 212 participants
• Primary outcome timeframe: Study Visit 8 at 14 weeks
• Results posted on: 2022-03-22

Participant Flow

Participant milestones

Measure	FT218 Patients treated with FT218, once nightly sodium oxybate granules for oral suspension	Placebo Matching Placebo
Overall Study STARTED	107	105
Overall Study COMPLETED	69	79
Overall Study NOT COMPLETED	38	26

Reasons for withdrawal

Measure	FT218 Patients treated with FT218, once nightly sodium oxybate granules for oral suspension	Placebo Matching Placebo
Overall Study Protocol Violation	0	2
Overall Study Pregnancy	2	0
Overall Study Lack of Efficacy	2	8
Overall Study Adverse Event	21	3
Overall Study Withdrawal by Subject	11	11
Overall Study Lost to follow up	2	0
Overall Study Non Compliance with Investigational Product	0	1
Overall Study Excluded Concomitant Medication	0	1

Baseline Characteristics

Once-Nightly Sodium Oxybate for Treatment of Excessive Daytime Sleepiness and Cataplexy in Narcolepsy

Baseline characteristics by cohort

Measure	FT218 n=107 Participants Patients treated with FT218, once nightly sodium oxybate granules for oral suspension.	Placebo n=105 Participants Matching Placebo	Total n=212 Participants Total of all reporting groups
Age, Categorical <=18 years	9 Participants n=5 Participants	12 Participants n=7 Participants	21 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	97 Participants n=5 Participants	91 Participants n=7 Participants	188 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants
Age, Continuous	30.9 years STANDARD_DEVIATION 10.70 • n=5 Participants	31.6 years STANDARD_DEVIATION 11.24 • n=7 Participants	31.2 years STANDARD_DEVIATION 10.95 • n=5 Participants
Sex: Female, Male Female	69 Participants n=5 Participants	75 Participants n=7 Participants	144 Participants n=5 Participants
Sex: Female, Male Male	38 Participants n=5 Participants	30 Participants n=7 Participants	68 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	8 Participants n=7 Participants	11 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	21 Participants n=5 Participants	15 Participants n=7 Participants	36 Participants n=5 Participants
Race (NIH/OMB) White	80 Participants n=5 Participants	80 Participants n=7 Participants	160 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Region of Enrollment United States	63 Participants n=5 Participants	53 Participants n=7 Participants	116 Participants n=5 Participants
Region of Enrollment Australia	4 Participants n=5 Participants	7 Participants n=7 Participants	11 Participants n=5 Participants
Region of Enrollment Canada	28 Participants n=5 Participants	31 Participants n=7 Participants	59 Participants n=5 Participants
Region of Enrollment Czechia	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment Denmark	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Region of Enrollment France	3 Participants n=5 Participants	5 Participants n=7 Participants	8 Participants n=5 Participants
Region of Enrollment Netherlands	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Region of Enrollment United Kingdom	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants

PRIMARY outcome

Timeframe: Study Visit 8 at 14 weeks

Population: Maintenance of Wakefulness Test (MWT) Mean Sleep Latency (Minutes) Change from Baseline to the End of the 9.0g Treatment Period - MMRM (mixed model repeat measure) Analysis (mITT Population)

Change from Baseline for MWT, which is the mean latency across 5 naps, averaged over the test day

Outcome measures

Measure	FT218 n=68 Participants Patients treated with FT218, once nightly sodium oxybate granules for oral suspension	Placebo n=78 Participants Matching Placebo
Maintenance of Wakefulness Test (MWT)	10.8 minutes Standard Deviation 0.96	4.7 minutes Standard Deviation 0.92

PRIMARY outcome

Timeframe: Study Visit 8 at 14 weeks

Population: Clinical Global Impression - Improvement (CGI-I) by the End of 9.0g Treatment Period

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 measures

Measure	FT218 n=69 Participants Patients treated with FT218, once nightly sodium oxybate granules for oral suspension	Placebo n=79 Participants Matching Placebo
Proportion of Patients That Were Very Much Improved or Much Improved on Clinical Global Impression of Improvement as Compared to Screening	72.0 percentage of subjects	31.6 percentage of subjects

PRIMARY outcome

Timeframe: Visit 8 - Change from Baseline at 14 Weeks

Population: Mean Weekly Number of Cataplexy Attacks (NCA) of Each Dosing Period, Change from Baseline to the End of 9.0g Treatment Period - MMRM Analysis (NT1 Subjects in mITT Population)

Mean number of cataplexy events recorded on the Sleep and Symptom Daily Diary during the period

Outcome measures

Measure	FT218 n=54 Participants Patients treated with FT218, once nightly sodium oxybate granules for oral suspension	Placebo n=62 Participants Matching Placebo
Number of Cataplexy Attacks at Visit 8 (Week 14) as Compared to Baseline	-11.5 Cataplexy Attacks Standard Deviation 0.96	-4.9 Cataplexy Attacks Standard Deviation 0.95

Adverse Events

FT218 9g Group

Serious events: 1 serious events

Other events: 23 other events

Deaths: 0 deaths

Placebo 9g Group

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Measure	FT218 9g Group n=77 participants at risk Patients treated with FT218 who completed the 9g dosing period	Placebo 9g Group n=80 participants at risk Patients treated with FT218 who completed the 9g dosing period
Psychiatric disorders Suicidal ideation	1.3% 1/77 • Number of events 1 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.

Other adverse events

Measure	FT218 9g Group n=77 participants at risk Patients treated with FT218 who completed the 9g dosing period	Placebo 9g Group n=80 participants at risk Patients treated with FT218 who completed the 9g dosing period
Nervous system disorders Dizziness	5.2% 4/77 • Number of events 4 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Nervous system disorders Somnolence	3.9% 3/77 • Number of events 3 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Psychiatric disorders Enuresis	9.1% 7/77 • Number of events 9 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Gastrointestinal disorders Vomiting	5.2% 4/77 • Number of events 5 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Metabolism and nutrition disorders Decreased Appetite	2.6% 2/77 • Number of events 2 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Metabolism and nutrition disorders Weight Decreased	3.9% 3/77 • Number of events 3 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	0.00% 0/80 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Nervous system disorders Headache	3.9% 3/77 • Number of events 3 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	3.8% 3/80 • Number of events 3 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Gastrointestinal disorders Nausea	3.9% 3/77 • Number of events 3 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	1.2% 1/80 • Number of events 2 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.
Infections and infestations Nasopharyngitis	2.6% 2/77 • Number of events 2 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.	2.5% 2/80 • Number of events 2 • AE (adverse event) data were obtained at all study visits (scheduled or unscheduled) from the time of the signing of informed consent until seven days after the last dose of study drug, at 14 weeks.

Additional Information

Jennifer Gudeman

Flamel Ireland Limited

Phone: +1 (314) 750-2751

Email: jgudeman@avadel.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place