Trial Outcomes & Findings for A Novel Pharmacological Therapy for Obstructive Sleep Apnea (NCT NCT03919955)

NCT ID: NCT03919955

Last Updated: 2025-12-23

Results Overview

Apneas and hypopneas per hour (3% desat and/or arousal), % change from baseline

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

117 participants

Primary outcome timeframe

one month

Results posted on

2025-12-23

Participant Flow

117 were enrolled, 58 passed baseline screening and were randomized.

Participant milestones

Participant milestones
Measure	Sequence AB Patients randomized to Placebo then Active	Sequence BA Patients randomized to Placebo then Active
Overall Study STARTED	29	29
Overall Study Completed Treatment Period 1	26	27
Overall Study Started Treatment Period 2	26	26
Overall Study COMPLETED	26	24
Overall Study NOT COMPLETED	3	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Novel Pharmacological Therapy for Obstructive Sleep Apnea

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=58 Participants All participants
Age, Continuous	52 Years n=68 Participants
Sex: Female, Male Female	20 Participants n=68 Participants
Sex: Female, Male Male	38 Participants n=68 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=68 Participants
Race (NIH/OMB) Asian	11 Participants n=68 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=68 Participants
Race (NIH/OMB) Black or African American	9 Participants n=68 Participants
Race (NIH/OMB) White	35 Participants n=68 Participants
Race (NIH/OMB) More than one race	2 Participants n=68 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=68 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	50 Participants n=68 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=68 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	3 Participants n=68 Participants

PRIMARY outcome

Timeframe: one month

Population: Population reported here is the population who completed a given treatment period. (Rather, formal statistical analysis was performed using multiple imputation.)

Apneas and hypopneas per hour (3% desat and/or arousal), % change from baseline

Outcome measures

Outcome measures
Measure	Active Intervention n=50 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Apnea-hypopnea Index [AHI]	-11.8 events/hr; % change from baseline Interval -44.9 to 7.2	-2.3 events/hr; % change from baseline Interval -29.3 to 17.6

SECONDARY outcome

Timeframe: one month

Population: secondary outcomes are presented as per primary outcome

Desaturation area under curve × event frequency, % change from baseline

Outcome measures

Outcome measures
Measure	Active Intervention n=50 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Hypoxic Burden	-15.0 %.min/hr; % change from baseline Interval -46.9 to 22.3	-8.7 %.min/hr; % change from baseline Interval -26.9 to 19.9

SECONDARY outcome

Timeframe: one month

Scored EEG arousals per hour (\>3 s), % change from baseline

Outcome measures

Outcome measures
Measure	Active Intervention n=50 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Arousal Index	-0.3 events/hr; % change from baseline Interval -23.9 to 41.5	-3.7 events/hr; % change from baseline Interval -26.2 to 22.2

SECONDARY outcome

Timeframe: one month

Population: All participants who completed questionnaires on treatment.

Self-reported sleepiness on scale of 0-24, higher values indicated greater sleepiness

Outcome measures

Outcome measures
Measure	Active Intervention n=51 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Epworth Sleepiness Scale	0 units on a scale; change from baseline Interval -3.0 to 2.0	-1 units on a scale; change from baseline Interval -3.0 to 2.0

SECONDARY outcome

Timeframe: one month

Disease-specific daytime function on scale of 5-20, higher values indicate greater function and quality of life

Outcome measures

Outcome measures
Measure	Active Intervention n=51 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Functional Outcomes of Sleep Questionnaire, Short Form	0.33 units on a scale; change from baseline Interval -1.0 to 1.5	0 units on a scale; change from baseline Interval -1.0 to 2.0

SECONDARY outcome

Timeframe: one month

Disease-specific quality of life on scale of 1-7, higher values indicate greater sleep-apnea related quality of life

Outcome measures

Outcome measures
Measure	Active Intervention n=51 Participants Active Intervention	Placebo Intervention n=53 Participants Placebo intervention
Sleep Apnea Quality of Life Index, Short Form	0.3 units on a scale; change from baseline Interval -0.1 to 1.0	0.4 units on a scale; change from baseline Interval 0.0 to 1.1

Adverse Events

Active Intervention

Serious events: 1 serious events

Other events: 39 other events

Deaths: 0 deaths

Placebo Intervention

Serious events: 0 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Active Intervention n=55 participants at risk Active Intervention	Placebo Intervention n=55 participants at risk Placebo intervention
Gastrointestinal disorders Hospitalization and laparotomy for suspected bowel obstruction	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.

Other adverse events

Additional Information

Scott Sands

Brigham and Women's Hospital

Phone: 8579280341

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Active Intervention n=55 participants at risk Active Intervention	Placebo Intervention n=55 participants at risk Placebo intervention
Gastrointestinal disorders Dry mouth	49.1% 27/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	20.0% 11/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Drowsiness/Fatigue	23.6% 13/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	14.5% 8/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Insomnia	23.6% 13/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	3.6% 2/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Decreased appetite	14.5% 8/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Restlessness	14.5% 8/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Constipation	14.5% 8/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Nausea	12.7% 7/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	3.6% 2/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Dificulty urinating	10.9% 6/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Sexual problems / decreased libido	10.9% 6/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Abdominal discomfort / pain	9.1% 5/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Dry skin	7.3% 4/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Headache	7.3% 4/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Indigestion	7.3% 4/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Ear and labyrinth disorders Dizziness	5.5% 3/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	1.8% 1/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
General disorders Erectile dysfunction	5.5% 3/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Vomiting	5.5% 3/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.
Gastrointestinal disorders Difficulty swallowing	5.5% 3/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.	0.00% 0/55 • One month Systematically documented per checklist, plus additional space to note new AEs. Subclassified as mild, moderate, or severe by self-report.