A Phase 2 interventional study of Topical acetyl hexapeptide-8 and Placebo in Focal Dystonia, sponsored by National Institute of Neurological Disorders and Stroke (NINDS). Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2016-11-10.
Sponsored by National Institute of Neurological Disorders and Stroke (NINDS) · Phase 2, Interventional, and Treatment
Background:
Objectives:
Eligibility:
Design:
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:
Non-parametric comparison (Wilcoxon Mann-Whitney test) of the primary and secondary variables before and after the intervention will be used for statistical analysis. Additional multivariate analysis will explore the influence of other factors (age, gender, disease duration).
Interventions and Duration
Twice daily application of active substance or placebo. Two active substance concentrations, 0.025% and 0.05% AH-8 will be used.
Each subject will be in the study a total of at least 3 months, followed by additional up to 4 months in the second phase for the group 2 patients.
Sample Size and Population
There will be 8 patients per arm, for a total study population of 24. The power calculation for a power of 80%, for a 2-tailed alpha of 0.05, to be able to detect a change of 2 points on the JBRS requires 8 patients per arm. This calculation was based on independent calculation for each dose of the active agent against placebo.
The subjects included will be patients with primary blepharospasm, not currently treated with BoNT (de novo diagnosed patients), but who subjectively express a wish for treatment intervention, with at least one year history from symptom onset, without perceived active change in symptoms by history. Patients with blepharospasm as part of a generalized dystonia or due to a different neurologic condition will be excluded.
Randomization and blinding to be performed by the NIH research pharmacy.
95 studies on the registry are indexed under Dystonic Disorders; 19 are open to participants now.
This study's enrollment of 8 is below the median of 24 across 45 interventional studies indexed under Dystonic Disorders.
Browse Dystonic Disorders studies →National Institute of Neurological Disorders and Stroke (NINDS) is the lead sponsor of 601 studies on the registry; 56 are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 8 (44%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Individuals off BoNT therapy for at least 4 months will be eligible for this study.
EXCLUSION CRITERIA:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 brand name, Argireline.
Drug: Topical acetyl hexapeptide-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 brand name, Argireline.
Drug: Topical acetyl hexapeptide-8
Participants in the Placebo arm received the placebo.
Drug: Placebo
The Jankovic Blepharospasm Rating Scale at 2 Month
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.
Time frame: 2 months
The Jankovic Blepharospasm Rating Scale at 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.
Time frame: 1 month
The Blepharospasm Disability Scale at 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.
Time frame: 1 month
The Blepharospasm Disability Scale at 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.
Time frame: 2 months
The Jankovic Blepharospasm Rating Scale at 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.
Time frame: 3 months
The Jankovic Blepharospasm Rating Scale at 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.
Time frame: 6 months
The Jankovic Blepharospasm Rating Scale at 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.
Time frame: 7 months
| Milestone | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| Started | 3 | 2 | 3 |
| Completed | 2 | 2 | 3 |
| Not completed | 1 | 0 | 0 |
| Withdrew: Adverse event | 1 | 0 | 0 |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Jankovic Blepharospasm Rating Scale at 2 Month | 6 (6 to 6) | 7 (6 to 8) | 4.33 (2 to 6) |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Jankovic Blepharospasm Rating Scale at 1 Month | 6.66 (6 to 8) | 5.5 (4 to 7) | 5 (2 to 7) |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Blepharospasm Disability Scale at 1 Month | 16.33 (14 to 20) | 16.5 (16 to 17) | 14.66 (10 to 18) |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Blepharospasm Disability Scale at 2 Months | 18.5 (17 to 20) | 15 (12 to 18) | 13.33 (8 to 16) |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Jankovic Blepharospasm Rating Scale at 3 Months | 6.5 (6 to 7) | 3 (2 to 4) | 2.33 (1 to 4) |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Jankovic Blepharospasm Rating Scale at 6 Months | 8 | 6 | 0 |
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.
| units on a scale | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| The Jankovic Blepharospasm Rating Scale at 7 Months | 8 | — | 2 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| 0.05% AH-8 | — | 1/3 (33.3%) | 2/3 (66.7%) |
| 0.025% AH-8 | — | 0/2 (0%) | 0/2 (0%) |
| Placebo | — | 1/3 (33.3%) | 2/3 (66.7%) |
| Event | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| Automobile accidentGeneral disorders | 1/3 | 0/2 | 0/3 |
| BlepharitisEye disorders | 0/3 | 0/2 | 1/3 |
| Event | 0.05% AH-8 | 0.025% AH-8 | Placebo |
|---|---|---|---|
| Puffiness of lower eyelidEye disorders | 1/3 | 0/2 | 0/3 |
| High blood pressure due to not taking blood pressure medicationCardiac disorders | 1/3 | 0/2 | 0/3 |
| Tingling in bilateral eyelids after cream applicationEye disorders | 0/3 | 0/2 | 1/3 |
| Weakness following botulinum toxin injectionEye disorders | 0/3 | 0/2 | 1/3 |
| Age, Categorical(Participants) | 0.05% AH-8 | 0.025% AH-8 | Placebo | Total |
|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 2 | 2 | 1 | 5 |
| >=65 years | 1 | 0 | 2 | 3 |
| Sex: Female, Male(Participants) | 0.05% AH-8 | 0.025% AH-8 | Placebo | Total |
|---|---|---|---|---|
| Female | 1 | 2 | 3 | 6 |
| Male | 2 | 0 | 0 | 2 |
| Jankovic Blepharospasm Rating Scale(units on a scale) | 0.05% AH-8 | 0.025% AH-8 | Placebo | Total |
|---|---|---|---|---|
| Mean | 7 (6 to 8) | 6 (6 to 6) | 6 (5 to 7) | 6.375 (5 to 8) |
| Blepharospasm Disability Scale(units on a scale) | 0.05% AH-8 | 0.025% AH-8 | Placebo | Total |
|---|---|---|---|---|
| Mean | 18 (16 to 21) | 16 (15 to 17) | 17.33 (16 to 19) | 17.25 (15 to 21) |
The record gives a start of Nov 2012, a primary completion of May 2015 and a completion of May 2015. It does not say whether these dates are actual or estimated.
Results were first posted in Nov 2016, 1 year 6 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Sep 2016. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
National Institute of Neurological Disorders and Stroke (NINDS) →