Trial Outcomes & Findings for Acetyl Hexapeptide-8 for Blepharospasm (NCT NCT01750346)

NCT ID: NCT01750346

Last Updated: 2016-11-10

Results Overview

The Jankovic Blepharospasm Rating Scale (JBRS) at 2 months from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 8 participants
• Primary outcome timeframe: 2 months
• Results posted on: 2016-11-10

Participant Flow

Participant milestones

Measure	0.05% AH-8 Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo Participants in the Placebo arm received the placebo.
Overall Study STARTED	3	2	3
Overall Study COMPLETED	2	2	3
Overall Study NOT COMPLETED	1	0	0

Reasons for withdrawal

Measure	0.05% AH-8 Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo Participants in the Placebo arm received the placebo.
Overall Study Adverse Event	1	0	0

Baseline Characteristics

Acetyl Hexapeptide-8 for Blepharospasm

Baseline characteristics by cohort

Measure	0.05% AH-8 n=3 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.	Total n=8 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants	2 Participants n=7 Participants	1 Participants n=5 Participants	5 Participants n=4 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants	3 Participants n=4 Participants
Sex: Female, Male Female	1 Participants n=5 Participants	2 Participants n=7 Participants	3 Participants n=5 Participants	6 Participants n=4 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	2 Participants n=4 Participants
Jankovic Blepharospasm Rating Scale	7 units on a scale n=5 Participants	6 units on a scale n=7 Participants	6 units on a scale n=5 Participants	6.375 units on a scale n=4 Participants
Blepharospasm Disability Scale	18 units on a scale n=5 Participants	16 units on a scale n=7 Participants	17.33 units on a scale n=5 Participants	17.25 units on a scale n=4 Participants

PRIMARY outcome

Timeframe: 2 months

Population: One participant in the 0.05% AH-8 arm was withdrawn due to development of blepharitis.

The Jankovic Blepharospasm Rating Scale (JBRS) at 2 months from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

Outcome measures

Measure	0.05% AH-8 n=2 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.
The Jankovic Blepharospasm Rating Scale at 2 Month	6 units on a scale Interval 6.0 to 6.0	7 units on a scale Interval 6.0 to 8.0	4.33 units on a scale Interval 2.0 to 6.0

SECONDARY outcome

Timeframe: 1 month

The Jankovic Blepharospasm Rating Scale (JBRS) was measured at 1 month from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

Outcome measures

Measure	0.05% AH-8 n=3 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.
The Jankovic Blepharospasm Rating Scale at 1 Month	6.66 units on a scale Interval 6.0 to 8.0	5.5 units on a scale Interval 4.0 to 7.0	5 units on a scale Interval 2.0 to 7.0

SECONDARY outcome

Timeframe: 1 month

The Blepharospasm Disability Scale (BDS) was measured at 1 month from the start of the study drug or placebo. The BDS is a scale which measures the impact of blepharospasm on the activities of daily living, i.e., need to wear sunglasses (1 or 2) and the impact on the following activities: driving (1 to 5), reading (1 to 3), watching tv (1 to 3), watching movies (1 to 3), shopping (1 to 3), walking about (1 to 4) and housework or job (1 to 3). Patients self-report disability in all areas for a total score between 0 to 26, where 0 indicates no symptoms and 26 indicates severe disability.

Outcome measures

Measure	0.05% AH-8 n=3 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.
The Blepharospasm Disability Scale at 1 Month	16.33 units on a scale Interval 14.0 to 20.0	16.5 units on a scale Interval 16.0 to 17.0	14.66 units on a scale Interval 10.0 to 18.0

SECONDARY outcome

Timeframe: 2 months

The Blepharospasm Disability Scale (BDS) was measured at 2 months from the start of the study drug or placebo. The BDS is a scale which measures the impact of blepharospasm on the activities of daily living, i.e., need to wear sunglasses (1 or 2) and the impact on the following activities: driving (1 to 5), reading (1 to 3), watching tv (1 to 3), watching movies (1 to 3), shopping (1 to 3), walking about (1 to 4) and housework or job (1 to 3). Patients self-report disability in all areas for a total score between 0 to 26, where 0 indicates no symptoms and 26 indicates severe disability.

Outcome measures

Measure	0.05% AH-8 n=2 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.
The Blepharospasm Disability Scale at 2 Months	18.5 units on a scale Interval 17.0 to 20.0	15 units on a scale Interval 12.0 to 18.0	13.33 units on a scale Interval 8.0 to 16.0

SECONDARY outcome

Timeframe: 3 months

The Jankovic Blepharospasm Rating Scale (JBRS) was measured at 3 months from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

Outcome measures

Measure	0.05% AH-8 n=2 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=2 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=3 Participants Participants in the Placebo arm received the placebo.
The Jankovic Blepharospasm Rating Scale at 3 Months	6.5 units on a scale Interval 6.0 to 7.0	3 units on a scale Interval 2.0 to 4.0	2.33 units on a scale Interval 1.0 to 4.0

SECONDARY outcome

Timeframe: 6 months

The Jankovic Blepharospasm Rating Scale (JBRS) was measured at 6 months from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

Outcome measures

Measure	0.05% AH-8 n=1 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 n=1 Participants Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=1 Participants Participants in the Placebo arm received the placebo.
The Jankovic Blepharospasm Rating Scale at 6 Months	8 units on a scale	6 units on a scale	0 units on a scale

SECONDARY outcome

Timeframe: 7 months

The Jankovic Blepharospasm Rating Scale (JBRS) was measured at 7 months from the start of the study drug or placebo. The JBRS is a clinical scale measuring the severity and frequency of blepharospasm. Severity and frequency are rated on a 5-point scales ranging from 0 to 4, where 0 indicates no symptoms and 4 indicates the most severe or frequent symptoms (Severity: severe, incapacitating spasm of eyelids and possibly other facial muscles; Frequency: Functionally "blind" due to persistent eye closure (blepharospasm) more than 50% of the waking time.) A total score is obtained by adding the severity and frequency subscale scores with total scores ranging from 0 to 8.

Outcome measures

Measure	0.05% AH-8 n=1 Participants Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	0.025% AH-8 Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline.	Placebo n=1 Participants Participants in the Placebo arm received the placebo.
The Jankovic Blepharospasm Rating Scale at 7 Months	8 units on a scale	—	2 units on a scale

Adverse Events

0.05% AH-8

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

0.025% AH-8

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Measure	0.05% AH-8 n=3 participants at risk Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline. Topical acetyl hexapeptide-8	0.025% AH-8 n=2 participants at risk Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline. Topical acetyl hexapeptide-8	Placebo n=3 participants at risk Participants in the Placebo arm received the placebo. Topical acetyl hexapeptide-8
General disorders Automobile accident	33.3% 1/3 • Number of events 1	0.00% 0/2	0.00% 0/3
Eye disorders Blepharitis	0.00% 0/3	0.00% 0/2	33.3% 1/3 • Number of events 1

Other adverse events

Measure	0.05% AH-8 n=3 participants at risk Participants in the 0.05% AH-8 arm received the higher dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline. Topical acetyl hexapeptide-8	0.025% AH-8 n=2 participants at risk Participants in the 0.025% AH-8 arm received the lower dose of the study drug, Acetyl Hexapeptide 8 (AH-8), which is sold under the band name, Argireline. Topical acetyl hexapeptide-8	Placebo n=3 participants at risk Participants in the Placebo arm received the placebo. Topical acetyl hexapeptide-8
Eye disorders Puffiness of lower eyelid	33.3% 1/3 • Number of events 1	0.00% 0/2	0.00% 0/3
Cardiac disorders High blood pressure due to not taking blood pressure medication	33.3% 1/3 • Number of events 1	0.00% 0/2	0.00% 0/3
Eye disorders Tingling in bilateral eyelids after cream application	0.00% 0/3	0.00% 0/2	33.3% 1/3 • Number of events 1
Eye disorders Weakness following botulinum toxin injection	0.00% 0/3	0.00% 0/2	33.3% 1/3 • Number of events 1

Additional Information

Dr. Codrin Lungu

National Institutes of Health

Phone: 301-402-0976

Email: lunguci@ninds.nih.gov

Results disclosure agreements

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