Focal DystoniaPhase 1/2NCT00942851
A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm
A Phase 1/2 study testing Acetyl-Hexapeptide Topical Treatment in people with Focal Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Focal Dystonia
- What is being tested?
- Acetyl-Hexapeptide Topical Treatment
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2009to Oct 2010
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age above 18 years
- Diagnosis of PB made by a Movement Disorders Neurologist
- Severity of PB prompting the need for treatment as determined by patient subjective assessment.
- JBRS of at least 3 at initial visit.
- BDS of at least 8 at the entry visit.
- At least three prior successful injectable BoNT treatments, with stable interval between injections of 3 months (ie duration of response of 3 months).
Who cannot join
- Pregnant women
- 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
- Skin condition resulting in loss of integrity of the skin overlying the OO or potentially interfering with cream absorption.
- Medical condition impairing the patient's ability to comply with the study protocol or to perform daily applications of the cream as instructed
- 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.
- Allergy to any component of the study or placebo cream.
- Known or observed eye pathology.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
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.
Read more
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.
Read the 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
- Conditions
- Focal Dystonia
- Being tested
- Acetyl-Hexapeptide Topical Treatment, placebo
- Keywords
- Dystonia, Blepharospasm, Botulinum Toxin, Treatment, Clinical Trial, Focal Dystonia
- Medical subject headings
- Dystonic Disorders, Dystonia, Blepharospasm
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acetyl-Hexapeptide Topical TreatmentDrug
AH-8 containing topical treatment
placeboDrug
topical treatment NOT containing AH-8
| Study group | Role | What they receive |
|---|---|---|
| active | Experimental | AH-8 containing topical intervention Receives: Drug: Acetyl-Hexapeptide Topical Treatment |
| placebo | Placebo comparator | topical intervention WITHOUT AH-8 Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time Until Jankovic Blepharospasm Rating Scale (JBRS) Reverts Back to Baseline
The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time. Return to baseline value represents loss of benefit from BoNT injection. The time to return to baseline JBRS is an indication of added benefit from the topical intervention.
Measured: 3-7 months
Secondary outcomes (2)
Change in the JBRS at 3 Months
The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time.
Measured: baseline to 3 months
% Blepharospasm Disability Scale (BDS) Change at 3 Months
% BDS change at 3 months. The BDS is a quality of life scale ranging from 0 (no symptoms) to 26 (maximum impact of symptoms on quality of life). The questions are specific for impairment related to blepharospasm. We compared the percentage change over three months in the active and placebo arms.
Measured: baseline to 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2009 (estimated)
- Study startJul 2009
- Primary completionOct 2010
- Study completionOct 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Focal DystoniaStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Collaborators
- BCN Peptides
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00942851
- Sponsor's protocol ID
- 090193
- Other IDs
- 09-N-0193
- Status last verified
- Aug 2012
- First submitted
- 18 Jul 2009
- Last updated
- 6 Sep 2012
- Results posted
- Yes, on ClinicalTrials.gov
Publications
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.
PubMed 15970766Defazio G, Livrea P. Primary blepharospasm: diagnosis and management. Drugs. 2004;64(3):237-44. doi: 10.2165/00003495-200464030-00002.
PubMed 14871168Defazio G, Livrea P. Epidemiology of primary blepharospasm. Mov Disord. 2002 Jan;17(1):7-12. doi: 10.1002/mds.1275.
PubMed 11835433
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study of Acetyl Hexapeptide-8 (AH8) in Treatment of Blepharospasm
- ClinicalTrials.gov ID
- NCT00942851
- Acronym / sponsor ID
- 090193
- Conditions
- Focal Dystonia
- Keywords
- Dystonia, Blepharospasm, Botulinum Toxin, Treatment, Clinical Trial, Focal Dystonia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Collaborators
- BCN Peptides
- Study start
- Jul 2009
- Primary completion
- Oct 2010 (actual)
- Study completion
- Oct 2010 (actual)
- First posted
- 21 Jul 2009 (estimated)
- Results
- Posted 6 Sep 2012
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| active | Experimental | Drug: Acetyl-Hexapeptide Topical TreatmentAH-8 containing topical intervention |
| placebo | Placebo comparator | Drug: placebotopical intervention WITHOUT AH-8 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
Key exclusion criteria (7 listed)
- Pregnant women
- 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
- Skin condition resulting in loss of integrity of the skin overlying the OO or potentially interfering with cream absorption.
- Medical condition impairing the patient's ability to comply with the study protocol or to perform daily applications of the cream as instructed
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Acetyl-Hexapeptide Topical TreatmentAH-8 containing topical treatment | Drug | active |
| placebotopical treatment NOT containing AH-8 | Drug | placebo |
Primary outcome
Time Until Jankovic Blepharospasm Rating Scale (JBRS) Reverts Back to Baseline
Time frame: 3-7 months
The JBRS is a severity scale for blepharospasm, ranging from 0 (no symptoms) to 8 (the most severe symptoms, functionally blind). It includes measures of blink frequency and severity of abnormal eye closure. The scale was measured at baseline (before intervention) and followed over time. Return to baseline value represents loss of benefit from BoNT injection. The time to return to baseline JBRS is an indication of added benefit from the topical intervention.
2 secondary outcomes
Change in the JBRS at 3 Months
Time frame: baseline to 3 months
% Blepharospasm Disability Scale (BDS) Change at 3 Months
Time frame: baseline to 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2012.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 15970766Ben 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.
- Background: PubMed 14871168Defazio G, Livrea P. Primary blepharospasm: diagnosis and management. Drugs. 2004;64(3):237-44. doi: 10.2165/00003495-200464030-00002.
- Background: PubMed 11835433Defazio G, Livrea P. Epidemiology of primary blepharospasm. Mov Disord. 2002 Jan;17(1):7-12. doi: 10.1002/mds.1275.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Acetyl-Hexapeptide Topical Treatment in people with Focal Dystonia, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Sep 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Sep 2012). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Time Until Jankovic Blepharospasm Rating Scale (JBRS) Reverts Back to Baseline (time frame: 3-7 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00942851, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Sep 2012.