Trial Outcomes & Findings for A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm (NCT NCT00942851)

Results Overview

The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time. Return to baseline value represents loss of benefit from BoNT injection. The time to return to baseline JBRS is an indication of added benefit from the topical intervention.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

24 participants

Primary outcome timeframe

3-7 months

Results posted on

2012-09-06

Participant Flow

Participant milestones

Participant milestones
Measure	Active Topical intervention agent containing AH8 0.005% Twice daily application to the eyelids in standardized fashion.	Placebo Topical intervention agent WITHOUT AH-8. Identically appearing cream without the active ingredient. Twice daily application to the eyelids in standardized fashion.
Overall Study STARTED	12	12
Overall Study COMPLETED	12	11
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Active n=12 Participants AH-8 containing topical intervention	Placebo n=12 Participants topical intervention WITHOUT AH-8	Total n=24 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
Age, Categorical Between 18 and 65 years	10 Participants n=5 Participants	11 Participants n=7 Participants	21 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Age Continuous	57.59 years STANDARD_DEVIATION 8.46 • n=5 Participants	55.87 years STANDARD_DEVIATION 8.25 • n=7 Participants	56.73 years STANDARD_DEVIATION 8.22 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants	2 Participants n=7 Participants	14 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants	10 Participants n=7 Participants	10 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants	12 participants n=7 Participants	24 participants n=5 Participants

PRIMARY outcome

Timeframe: 3-7 months

The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time. Return to baseline value represents loss of benefit from BoNT injection. The time to return to baseline JBRS is an indication of added benefit from the topical intervention.

Outcome measures

Outcome measures
Measure	Active Ingredient- AH8 n=12 Participants subjects receiving active intervention, ie topical cream containing 0.005% AH-8	Placebo n=11 Participants subjects receiving placebo intervention, ie identically-appearing topical cream without AH-8 content
Time Until Jankovic Blepharospasm Rating Scale (JBRS) Reverts Back to Baseline	3.71 month Standard Deviation 1.48	3.03 month Standard Deviation .23

SECONDARY outcome

Timeframe: baseline to 3 months

Population: One placebo arm participant dropped out due to unrelated personal problems

The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time.

Outcome measures

Outcome measures
Measure	Active Ingredient- AH8 n=12 Participants subjects receiving active intervention, ie topical cream containing 0.005% AH-8	Placebo n=11 Participants subjects receiving placebo intervention, ie identically-appearing topical cream without AH-8 content
Change in the JBRS at 3 Months	1.09 points Standard Deviation 0.7	0.92 points Standard Deviation 2.07

SECONDARY outcome

Timeframe: baseline to 3 months

Population: One placebo arm participant discontinued study due to unrelated personal problems

% BDS change at 3 months. The BDS is a quality of life scale ranging from 0 (no symptoms) to 26 (maximum impact of symptoms on quality of life). The questions are specific for impairment related to blepharospasm. We compared the percentage change over three months in the active and placebo arms.

Outcome measures

Outcome measures
Measure	Active Ingredient- AH8 n=12 Participants subjects receiving active intervention, ie topical cream containing 0.005% AH-8	Placebo n=11 Participants subjects receiving placebo intervention, ie identically-appearing topical cream without AH-8 content
% Blepharospasm Disability Scale (BDS) Change at 3 Months	30.89 percentage change Standard Deviation 37.3	32.32 percentage change Standard Deviation 31.13

Adverse Events

Active

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 2 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Active n=12 participants at risk AH-8 containing topical intervention	Placebo n=12 participants at risk topical intervention WITHOUT AH-8
Skin and subcutaneous tissue disorders eyelid irritation	16.7% 2/12 • Number of events 2	16.7% 2/12 • Number of events 2

Additional Information

Codrin Lungu, MD

Office of the Clinical Director, NINDS, NIH

Phone: 301-496-5295

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place