Clinical trial NCT00942851: A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm
Official title: A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Focal Dystonia
Keywords: Dystonia
Blepharospasm
Botulinum Toxin
Treatment
Clinical Trial
Focal Dystonia
Lead sponsor: National Institutes of Health Clinical Center (CC)
Sponsor class: NIH
Collaborators: name: BCN Peptides
First posted: 2009-07-21
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Background:

* Blepharospasm is caused by excessive contraction of the muscles that close the eye. It can be treated with injections of botulinum neurotoxin (BoNT), which works by weakening those muscles.
* Acetyl Hexapeptide-8 (AH-8) is the active ingredient in a number of cosmetic creams used to treat wrinkles, and is marketed under the trade name Argireline(Copyright). Like BoNT, AH-8 works to weaken the muscles, but is available as a skin cream instead of an injection. AH-8 has never been used to treat people with blepharospasm.

Objectives:

- To determine if AH-8 can be used as part of a treatment regimen for blepharospasm.

Eligibility:

- Individuals 18 years of age and older who have blepharospasm and have been receiving successful treatment with botulinum toxin injections.

Design:

* Participants will be involved in the study for a maximum of 7 months.
* Patients will have a complete physical and neurological exam, and will be asked questions about their blepharospasm. Patients will then receive BoNT injections in the same areas of the muscle around the eye and at the same doses that have been effective previously.
* After the injections, patients will receive a container of either the active cream (with AH-8) or cream without AH-8, and will be instructed on how to apply it.
* Patients will return 1 month after the first visit for another neurologic exam and questions, and will be asked about any side effects. Another supply of cream will be given.
* Five additional visits will take place on a monthly basis, and patients will be given additional supplies of the cream as needed. Patients will stop participating in the study if they require another BoNT injection for blepharospasm. The study will end after 7 months.

Detailed description: OBJECTIVE:

To study the efficacy of cutaneous application of Acetyl Hexapeptide-8 (AH8) in the therapy of blepharospasm.

STUDY POPULATION:

22 patients with blepharospasm.

DESIGN:

This will be a double blind, placebo-controlled trial. Patients receiving treatment of blepharospasm with botulinum neurotoxin (BoNT) will be recruited. They will receive either the study substance or a placebo cream containing the emulsion but no active substance, in twice daily applications to the eyelids beginning the day following a BoNT injection treatment. They will continue to apply the cream and we will record the time until their condition worsens back to baseline following benefit from the injections, hypothesizing that the cream application will prolong the time until the need for the next injection by at least 3 months.

OUTCOME MEASURES::

Primary: time until the Jankovic Blepharospasm Rating Scale (JBRS) reverts back to baseline.

Secondary: Change in the JBRS at 6 months; Change in the JBRS at 7 months if not receiving BoNT; Change in the JBRS at 3 months; Change in the Blepharospasm Disability Scale at 6 months

Eligibility criteria: * INCLUSION CRITERIA:

  1. Age above 18 years
  2. Diagnosis of PB made by a Movement Disorders Neurologist
  3. Severity of PB prompting the need for treatment as determined by patient subjective assessment.
  4. JBRS of at least 3 at initial visit.
  5. BDS of at least 8 at the entry visit.
  6. At least three prior successful injectable BoNT treatments, with stable interval between injections of 3 months (ie duration of response of 3 months).

EXCLUSION CRITERIA:

1. Pregnant women
2. Blepharospasm associated with a different Neurologic condition, including but not limited to generalyzed dystonia, a parkinsonian syndrome, or major brain structural abnormality, as evidenced by Neurologic exam and/or review of records
3. Skin condition resulting in loss of integrity of the skin overlying the OO or potentially interfering with cream absorption.
4. Medical condition impairing the patient's ability to comply with the study protocol or to perform daily applications of the cream as instructed
5. Abnormally long or short response to BoNT treatment in the past, ie duration of PB relief with BoNT greater than 4 months or less than 2 months.
6. Allergy to any component of the study or placebo cream.
7. Known or observed eye pathology.

Primary outcomes:
measure: Time Until Jankovic Blepharospasm Rating Scale (JBRS) Reverts Back to Baseline
timeFrame: 3-7 months
Secondary outcomes:
measure: Change in the JBRS at 3 Months
timeFrame: baseline to 3 months
measure: % Blepharospasm Disability Scale (BDS) Change at 3 Months
timeFrame: baseline to 3 months
Other outcomes:
Locations:
city: Bethesda
country: United States
facility: National Institutes of Health Clinical Center, 9000 Rockville Pike
state: Maryland
Study officials:
References:
citation: Ben Simon GJ, McCann JD. Benign essential blepharospasm. Int Ophthalmol Clin. 2005 Summer;45(3):49-75. doi: 10.1097/01.iio.0000167238.26526.a8. No abstract available.
citation: Defazio G, Livrea P. Primary blepharospasm: diagnosis and management. Drugs. 2004;64(3):237-44. doi: 10.2165/00003495-200464030-00002.
citation: Defazio G, Livrea P. Epidemiology of primary blepharospasm. Mov Disord. 2002 Jan;17(1):7-12. doi: 10.1002/mds.1275.