Clinical Trial: Acetyl Hexapeptide-8 for Blepharospasm
Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional
Official Title: Placebo Controlled Double Blind Study of Acetyl Hexapeptide-8 in Treatment of Blepharospasm
Brief Summary:
Background:
- Blepharospasm is caused by excessive contraction of the muscles that close the eye. One treatment is botulinum neurotoxin (BoNT), which works by weakening those muscles. Like BoNT, acetyl hexapeptide-8 (AH-8) works to weaken the muscles, but is available as a skin cream. AH-8 is the active ingredient in a number of cosmetic creams used to treat wrinkles. Researchers thought that AH-8 cream could be used to treat blepharospasm, but the original dose studied was not very effective. They want to try a higher dose of AH-8 in a cream to see if it can be a more effective treatment.
Objectives:
- To see if AH-8 cream can improve the symptoms of blepharospasm.
Eligibility:
- Individuals at least 18 years of age who have blepharospasm that is severe enough to require treatment.
Design:
- This study will involve up to eight study visits.
- Participants will be screened with a physical exam and medical history. They will answer questions about their symptoms. They will also have a blink test to see how severe the blepharospasm is. At this visit, participants will receive one of three types of cream. One cream will have a low dose of AH-8, one will have a higher dose of AH-8, and the other will be a placebo (no AH-8).
- One month later, participants will have a followup visit, with tests similar to the first visit. They will also receive more of the cream.
- One month later, participants will have another visit with the same tests. They will be vide
Detailed Summary:
OBJECTIVE:
Test the efficacy of topical AH-8 in blepharospasm treatment.
DESIGN:
This is a randomized, double blind, placebo-controlled study, phase II single center clinical trial. Patients with primary blepharospasm at least 4 months off BoNT therapy with no benefit from last BoNT injection by history and self assesment will be included. We will use 3 study arms placebo and 2 different doses (as concentrations of the AH-8 active substance in the topical preparation). An extension phase is planned after the initial phase.
The patients will have a baseline screening visit, including measurement of the outcome variables, then begin the study intervention. They will have 2 subsequent visits at month 1 and month 2. After this visit, the patients who do not have significant benefit and wish to have an alternative therapy (GROUP 1) will receive BoNT injections according to best practice standards. These patients will then be assessed 1 month later (peak effect of BoNT therapy) and the effect will be compared with the AH-8 effect. The patients who have significant benefit (GROUP 2) will continue therapy for another 4 months (total of 6 months), and will have another assessment at that point. Afterwards the patients will be offered BoNT injections according to best practice standards, similar to Group 1 above, and another assessment will be made one month later (month 7) to compare the effects with AH-8 effects.
OUTCOME MEASURES:
Primary outcome variable: the Jankovic Blepharospasm Rating Scale at month 2.
Secondary outcome variables:
Current Primary Outcome: The Jankovic Blepharospasm Rating Scale at 2 Month [ Time Frame: 2 months ]
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.
Original Primary Outcome: The Jankovic Blepharospasm Rating Scale at 2 Month [ Time Frame: 2 months ]
Current Secondary Outcome:
- The Jankovic Blepharospasm Rating Scale at 1 Month [ Time Frame: 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.
- The Blepharospasm Disability Scale at 1 Month [ Time Frame: 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.
- The Blepharospasm Disability Scale at 2 Months [ Time Frame: 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.
- The Jankovic Blepharospasm Rating Scale at 3 Months [ Time Frame: 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.
- The Jankovic Blepharospasm Rating Scale at 6 Months [ Time Frame: 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.
- The Jankovic Blepharospasm Rating Scale at 7 Months [ Time Frame: 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.
Original Secondary Outcome: JBRS at 1 mo; BDS at 1 and 2 mo; difference in JBRS between 2 and 3 months in group1; JBRS score at 6 months in group 2; different in JBRS between 6 and 7 months in group 2; blink reflex measures at 2 months [ Time Frame: 2 months ]
Information By: National Institutes of Health Clinical Center (CC)
Dates:
Date Received: December 12, 2012
Date Started: November 2012
Date Completion:
Last Updated: September 22, 2016
Last Verified: September 2016
- The Jankovic Blepharospasm Rating Scale at 1 Month [ Time Frame: 1 month ]
