Trial Outcomes & Findings for Efficacy and Safety of AUT00063 Versus Placebo in Age-Related Hearing Loss (NCT NCT02345031)

Last Updated: 2018-08-09

Results Overview

To compare the change in hearing using the QuickSIN test (speech in noise performance) from baseline (Day 1 to Day 28) between AUT00063 and placebo. The QuickSIN test measures the level of signal compared to the noise that is required to achieve 50% recognition. The test is administered in a sound booth at 70-dB HL binaurally via insert-ear phones. Three lists are administered to each individual subject and the threshold or 50% signal-to-noise ratio (SNR) is calculated as the mean of the three lists completed. Each list consists of six sentences with five key words to be scored per sentence. The sentences are presented in four-talker babble noise. The sentences are presented at pre-recorded signal-to-noise ratios which decrease in 5-dB steps from 25 (very easy) to 0 (extremely difficult).

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

78 participants

Primary outcome timeframe

28 days

Results posted on

2018-08-09

Participant Flow

Participant milestones

Participant milestones
Measure	AUT00063 (600 mg Capsules) 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Overall Study STARTED	38	40
Overall Study COMPLETED	37	39
Overall Study NOT COMPLETED	1	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Demographic data are listed from Full Analysis Set, excludes 2 subjects.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	AUT00063 (600 mg Capsules) n=38 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=40 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks	Total n=78 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	0 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	0 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.
Age, Categorical Between 18 and 65 years	15 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	13 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	28 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.
Age, Categorical >=65 years	22 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	26 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.	48 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects.
Sex: Female, Male Female	15 Participants n=37 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects	17 Participants n=39 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects	32 Participants n=76 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects
Sex: Female, Male Male	22 Participants n=37 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects	22 Participants n=39 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects	44 Participants n=76 Participants • Demographics data are listed from Full Analysis Set, excludes 2 subjects
Race/Ethnicity, Customized Race · White/Caucasian	36 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	38 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	74 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects
Race/Ethnicity, Customized Race · Black/African-American	1 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	1 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	2 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects
Race/Ethnicity, Customized Ethnicity · Hispanic Or Latino	1 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	0 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	1 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects
Race/Ethnicity, Customized Ethnicity · Not Hispanic Or Latino	36 Participants n=37 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	39 Participants n=39 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects	75 Participants n=76 Participants • Demographic data are listed from Full Analysis Set, excludes 2 subjects
Region of Enrollment United States	38 Participants n=38 Participants	40 Participants n=40 Participants	78 Participants n=78 Participants
QuickSIN SNR-50 Score (dB)	7.00 decibels (dB) STANDARD_DEVIATION 1.963 • n=37 Participants • QuickSIN data are listed from Full Analysis set, excludes 2 subjects	7.11 decibels (dB) STANDARD_DEVIATION 1.991 • n=39 Participants • QuickSIN data are listed from Full Analysis set, excludes 2 subjects	7.06 decibels (dB) STANDARD_DEVIATION 1.965 • n=76 Participants • QuickSIN data are listed from Full Analysis set, excludes 2 subjects

PRIMARY outcome

Timeframe: 28 days

Population: Full Analysis Set

Outcome measures

Outcome measures
Measure	AUT00063 (600 mg Capsules) n=37 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=39 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Change in Hearing Loss After 4 Weeks of Treatment	-1.114 decibels (dB) Interval -1.956 to -0.272	-1.914 decibels (dB) Interval -2.703 to -1.125

SECONDARY outcome

Timeframe: 28 days

Population: FAS Population

Final Average GDT Across-Channel at Day 28: Change from Baseline; FAS Population

Outcome measures

Outcome measures
Measure	AUT00063 (600 mg Capsules) n=35 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=37 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Analysis of Change From Baseline in Adaptive Test of Temporal Resolution (ATTR) on End-Point Visit Day 28	-2.978 ms Interval -17.57 to 11.612	-10.72 ms Interval -23.9 to 2.458

SECONDARY outcome

Timeframe: 28 days

Population: FAS Population

Final Average GDT Within-Channel at Day 28: Change from Baseline; FAS Population

Outcome measures

Outcome measures
Measure	AUT00063 (600 mg Capsules) n=36 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=39 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Analysis of Change From Baseline in Adaptive Test of Temporal Resolution (ATTR) on End-Point Visit Day 28	4.452 ms Interval 1.306 to 7.598	5.526 ms Interval 2.661 to 8.392

SECONDARY outcome

Timeframe: 42 Days

Number of Subjects With At Least One Treatment Emergent Adverse Event

Outcome measures

Outcome measures
Measure	AUT00063 (600 mg Capsules) n=38 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=40 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
To Further Investigate the Safety and Tolerability Profile of Repeat Administration of AUT00063 by Assessing Vital Signs, Physical Examination, Laboratory Exams and ECG	23 Participants	24 Participants

SECONDARY outcome

Timeframe: 28 Days

Exposure of AUT00063 ng/ml, in plasma levels at Day 28

Outcome measures

Outcome measures
Measure	AUT00063 (600 mg Capsules) n=36 Participants 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	(AUT00063 Placebo Capsules) n=36 Participants 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Pharmacokinetic of AUT00063, Plasma Levels	3470.61 ng Standard Deviation 1290.679	0 ng Standard Deviation 0

Adverse Events

AUT00063 (600 mg Capsules)

Serious events: 0 serious events

Other events: 18 other events

Deaths: 0 deaths

Placebo (AUT00063 Placebo Capsules)

Serious events: 1 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	AUT00063 (600 mg Capsules) n=38 participants at risk 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	Placebo (AUT00063 Placebo Capsules) n=40 participants at risk 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Injury, poisoning and procedural complications Alcohol poisoning	0.00% 0/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	2.5% 1/40 • Number of events 1 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.

Other adverse events

Other adverse events
Measure	AUT00063 (600 mg Capsules) n=38 participants at risk 3 capsules of 200 mg of the investigational drug AUT00063, to take orally once daily with food for 4 weeks AUT00063: 600 mg, orally, once a day, for 4 weeks	Placebo (AUT00063 Placebo Capsules) n=40 participants at risk 3 capsules of placebo, to take orally once daily with food for 4 weeks Placebo: orally, once a day, for 4 weeks
Musculoskeletal and connective tissue disorders Back Pain	2.6% 1/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	7.5% 3/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Investigations Blood triglycerides increased	5.3% 2/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	0.00% 0/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Gastrointestinal disorders Diarrhoea	7.9% 3/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	5.0% 2/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Nervous system disorders Dizziness	15.8% 6/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	2.5% 1/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
General disorders Fatigue	5.3% 2/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	0.00% 0/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Infections and infestations Gastroenteritis	0.00% 0/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	5.0% 2/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Nervous system disorders Headache	10.5% 4/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	12.5% 5/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Gastrointestinal disorders Nausea	5.3% 2/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	7.5% 3/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Respiratory, thoracic and mediastinal disorders Sinus Congestion	2.6% 1/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	5.0% 2/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Nervous system disorders Somnolence	7.9% 3/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	0.00% 0/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Ear and labyrinth disorders Tinnitus	10.5% 4/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	7.5% 3/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.
Infections and infestations Upper respiratory tract infection	7.9% 3/38 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.	5.0% 2/40 • The recording period for Adverse Events is the period starting from the Informed Consent signature until the safety close out visit or subject's study participation ends; a total duration of up to 10 weeks.

Additional Information

Clinical Project Manager

Autifony Therapeutics Ltd

Phone: +44 1438 906860

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60