Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting

Acetyl Hexapeptide-8 for Blepharospasm

This study has been terminated.
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) ) Identifier:
First received: December 12, 2012
Last updated: May 30, 2015
Last verified: May 2015


- 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.


- To see if AH-8 cream can improve the symptoms of blepharospasm.


- Individuals at least 18 years of age who have blepharospasm that is severe enough to require treatment.


  • 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.

Condition Intervention Phase
Focal Dystonia
Drug: Topical acetyl hexapeptide-8
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double-Blind
Primary Purpose: Treatment
Official Title: Placebo Controlled Double Blind Study of Acetyl Hexapeptide-8 in Treatment of Blepharospasm

Resource links provided by NLM:

Further study details as provided by National Institutes of Health Clinical Center (CC):

Primary Outcome Measures:
  • The Jankovic Blepharospasm Rating Scale at 2 month [ Time Frame: 2 months ]

Secondary Outcome Measures:
  • 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 ]

Enrollment: 8
Study Start Date: November 2012
Study Completion Date: May 2015
Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)
  Show Detailed Description


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • 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)


  • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01750346

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
Principal Investigator: Codrin I Lungu, M.D. National Institute of Neurological Disorders and Stroke (NINDS)
  More Information

Responsible Party: National Institute of Neurological Disorders and Stroke (NINDS) Identifier: NCT01750346     History of Changes
Other Study ID Numbers: 130022  13-N-0022 
Study First Received: December 12, 2012
Last Updated: May 30, 2015
Health Authority: United States: Federal Government

Keywords provided by National Institutes of Health Clinical Center (CC):

Additional relevant MeSH terms:
Dystonic Disorders
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Movement Disorders
Central Nervous System Diseases
Eyelid Diseases
Eye Diseases processed this record on October 21, 2016