Acetyl Hexapeptide-8 for Blepharospasm
Condition: Focal Dystonia
MeSH terms: Dystonia; Dystonic Disorders; Blepharospasm
Intervention: Topical acetyl hexapeptide-8; Placebo
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 8.0
Sex: All
Minimum age: 18 Years
Healthy volunteers: No
Keywords: Blepharospasm; Treatment; Dystonia; Acetyl-Hexapeptide

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
           videotaped at this visit to study their facial movements. Those who have responded to
           the treatment will continue to use the cream. Those who have not responded will be
           offered the chance to have BoNT injections, and will stop taking the cream.

        -  One month later, participants who had BoNT injections will have a final visit to check
           for possible side effects. Those who continued to take the cream will continue on the
           study.

        -  The fifth and sixth visits will involve the same tests as before. At the seventh visit,
           remaining participants will be offered the chance to have BoNT injections, and will stop
           taking the cream.

        -  The final visit will check for any side effects from the cream or the injections.
    

Eligibility criteria:

        -  INCLUSION CRITERIA:

          -  Primary blepharospasm diagnosed or confirmed by a Movement Disorders Neurologist,
             confirmed at the initial visit by a study investigator.

          -  Individuals off BoNT therapy for at least 4 months will be eligible for this study.

               -  Severity prompting need for treatment as determined by clinical judgment

               -  At least a score of 4 on the JBRS

               -  At least a score of 8 on the BDS

               -  Duration of symptoms of at least 1 year, without subjective active progression by
                  patient report

               -  Concomitant therapy allowed, except injectable BoNT therapy, provided the doses
                  remain stable throughout the study period

               -  Adult patients (> 18)

        EXCLUSION CRITERIA:

          -  Blepharospasm associated with generalized or extensive regional dystonia

          -  Medical condition impairing the patient's ability to comply with the study protocol or
             to perform daily applications of the cream as instructed as judged by recruiting
             physician

          -  Local eyelid pathology precluding topical treatment

          -  Received BoNT within 4 months prior to enrollment

          -  Continued benefit from a prior BoNT injection (by history and self assesment) Current
             use of cosmetic wrinkle creams

          -  Prior myectomy procedure excluded

          -  Pregnant women excluded. Barrier contraception will be used throughout the study for
             women of childbearing age, as it is not known how the use of hormonal contraceptives
             may interact with the study substance. Menopausal status will be determined by the CNS
             IRB criteria. In women of childbearing potential, a pregnancy test will be performed
             at the initial visit and periodically every 2 months for the duration of the study.
             Barrier contraception will be deemed necessary for the duration of the study and none
             after.

          -  Use of other treatments for blepharospasm allowed if the doses remain constant

          -  Allergy/sensitivity to study substance or vehicle.

          -  Active drug or alcohol abuse or dependence

          -  Patients with uncontrolled co-existing medical conditions: uncontrolled systemic
             hypertension with values above 170/100; active heart disease needing immediate
             intervention; active respiratory disease needing intervention; known or observed eye
             pathology; any condition that would render the patient unable to safely cooperate with
             the study tests as judged by the screening physician

          -  Cognitive inability to independently use cr(SqrRoot)(Registered Trademark)me use
             safely.