- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03286582
A Proof-of-Concept Study of Topical AC-203 in Patients With Bullous Pemphigoid
September 17, 2019 updated by: TWi Biotechnology, Inc.
A Randomized, Open-Label, Controlled Trial of Topical AC-203 in Subjects With Bullous Pemphigoid
Bullous pemphigoid (BP) is a chronic, inflammatory, subepidermal, autoimmune blistering disease which mainly develops in the elderly, with onset usually in the late 70s and a substantial increase in incidence in people older than 80 years.
If untreated, it can persist for months or years, with periods of spontaneous remissions and exacerbations.
It has been found that blisters and sera of BP patients contain abnormally high levels of pro-inflammatory cytokines such as interleukin-6 (IL-6) and IL-8.
Recently, it also has been demonstrated that NLRP3 (NACHT, LRR and PYD domains-containing protein 3) inflammasome components (the NLRP3-caspase-1-IL-18 axis) were significantly up-regulated in peripheral blood mononuclear cells from BP patients and positively correlated with disease activity.
AC-203 is a topical formulation of an oral modulator of inflammasome and IL-1beta pathways.
In vitro studies have demonstrated that AC-203 significantly reduced secretion of IL-6 and moderately reduced IL-8 secretion in HaCaT cells treated with specific anti-BP180 IgG.
This study is designed to test the safety, tolerability, efficacy, and pharmacokinetics of AC-203 ointment (vs. a topical steroid comparator representing standard of care) ointment in subjects with BP.
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
Study Type
Interventional
Enrollment (Actual)
10
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
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Taipei, Taiwan, 100
- National Taiwan University Hospital
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-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
20 years to 90 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- Age 20 to 90 years old, inclusive, at enrollment.
Diagnosis of bullous pemphigoid confirmed by histopathology and one of following assessments:
- Direct immunofluorescence (DIF)
- Indirect immunofluorescence (IIF)
- ELISA test (ELISA detection of immunoglobulin G (IgG) anti-BP180 autoantibodies in serum more than 9 U/mL).
- Localized or limited BP with the occurrence of <10 new blisters per day in the week prior to enrollment.
Is male, or is female and meets all the following criteria:
- Not breastfeeding
- If of childbearing potential (defined as non-post hysterectomy or non-post-menopausal [≥50 years of age and amenorrheic for at least 1 year]), must have a negative pregnancy test result (human chorionic gonadotropin, beta subunit [bhCG]) at Visit 1, and must practice and be willing to continue to practice appropriate birth control (abstinence, double barrier methods, hormonal contraceptives, intrauterine device, or tubal ligation) during the entire duration of the study
- Is able to understand and sign the Informed Consent Form (ICF), communicate with the investigator, and understand and comply with protocol requirements.
Exclusion Criteria:
- Diagnosis of pemphigus, dermatitis, eczema, psoriasis, or other skin condition which in the opinion of the investigator may confound diagnosis, treatment, or evaluation of bullous pemphigoid.
- Use of oral steroids in the 2 weeks prior to enrollment at a dose greater than prednisolone equivalent dose (PED) of 10 mg/day.
- Use of topical steroids for more than 3 consecutive days in the 2 weeks prior to enrollment.
- Use of non-steroid immunosuppressants including but not limited to azathioprine, mycophenolate, cyclophosphamide, chlorambucil, methotrexate, tacrolimus, or cyclosporine in the 2 weeks prior to enrollment.
- Use of systemic antibiotics in the 2 weeks prior to enrollment.
- Use of oral dapsone in the 2 weeks prior to enrollment.
- Treatment with intravenous immunoglobulin (IVIG) in the 8 weeks prior to enrollment.
- Any prior use of approved or investigational biologic anti-inflammatory therapy within 6 months prior to enrollment, including but not limited to: anakinra, rilonacept, canakinumab, etanercept, adalimumab, infliximab, rituximab, certolizumab, golimumab, tocilizumab, bertilimumab, or abatacept.
- Presence of active systemic infections.
- Any clinically significant medical condition or laboratory value that could potentially affect study participation and/or personal well-being, as judged by the investigator.
- History of allergy or hypersensitivity to any component of study medication or clobetasol.
- Has participated in a clinical study within 30 days prior to enrollment.
- Is an immediate family member (spouse, parent, child, or sibling; biological or legally adopted) of personnel directly affiliated with the study at the clinical study site, or is directly affiliated with the study at the clinical study site.
- Is employed by the sponsor (i.e., is an employee, temporary contract worker, or designee responsible for the conduct of the study).
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: AC-203
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AC-203 1% ointment BID (twice daily)
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Active Comparator: Clobetasol
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Clobetasol 0.05% Topical Ointment BID
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Incidence of adverse events during the treatment period
Time Frame: 10 Weeks
|
10 Weeks
|
Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Proportion achieving disease control (no new blisters within prior week)
Time Frame: 2, 4, 5, 6, 8, 10 Weeks
|
2, 4, 5, 6, 8, 10 Weeks
|
New blister count
Time Frame: 2, 4, 5, 6, 8, 10 Weeks
|
2, 4, 5, 6, 8, 10 Weeks
|
Time to disease control
Time Frame: 2, 4, 5, 6, 8, 10 Weeks
|
2, 4, 5, 6, 8, 10 Weeks
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Proportion of subjects who require rescue therapy prior to Week 6
Time Frame: 2, 4, 5, 6 Weeks
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2, 4, 5, 6 Weeks
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BPDAI (BP Disease Area Index) score
Time Frame: 2, 4, 5, 6, 8, 10 Weeks
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2, 4, 5, 6, 8, 10 Weeks
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Pruritus VAS (Visual Analogue Scale) score change from baseline
Time Frame: 2, 4, 5, 6, 8, 10 Weeks
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2, 4, 5, 6, 8, 10 Weeks
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DLQI (Dermatology Life Quality Index) score change from baseline
Time Frame: 6, 10 Weeks
|
6, 10 Weeks
|
Inflammation marker
Time Frame: 6 Weeks
|
6 Weeks
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 5, 2017
Primary Completion (Actual)
December 25, 2018
Study Completion (Actual)
January 22, 2019
Study Registration Dates
First Submitted
September 14, 2017
First Submitted That Met QC Criteria
September 14, 2017
First Posted (Actual)
September 18, 2017
Study Record Updates
Last Update Posted (Actual)
September 19, 2019
Last Update Submitted That Met QC Criteria
September 17, 2019
Last Verified
September 1, 2019
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AC-203-BP-001
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
No
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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