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A Proof-of-Concept Study of Topical AC-203 in Patients With Bullous Pemphigoid

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03286582
Recruitment Status : Terminated (Terminated with partial enrollment completed)
First Posted : September 18, 2017
Last Update Posted : September 19, 2019
Information provided by (Responsible Party):
TWi Biotechnology, Inc.

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Bullous Pemphigoid Drug: AC-203 1% Topical Ointment Drug: Clobetasol 0.05% Topical Ointment Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Randomized, Open-Label, Controlled Trial of Topical AC-203 in Subjects With Bullous Pemphigoid
Actual Study Start Date : September 5, 2017
Actual Primary Completion Date : December 25, 2018
Actual Study Completion Date : January 22, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Pemphigus

Arm Intervention/treatment
Experimental: AC-203 Drug: AC-203 1% Topical Ointment
AC-203 1% ointment BID (twice daily)

Active Comparator: Clobetasol Drug: Clobetasol 0.05% Topical Ointment
Clobetasol 0.05% Topical Ointment BID

Primary Outcome Measures :
  1. Incidence of adverse events during the treatment period [ Time Frame: 10 Weeks ]

Secondary Outcome Measures :
  1. Proportion achieving disease control (no new blisters within prior week) [ Time Frame: 2, 4, 5, 6, 8, 10 Weeks ]
  2. New blister count [ Time Frame: 2, 4, 5, 6, 8, 10 Weeks ]
  3. Time to disease control [ Time Frame: 2, 4, 5, 6, 8, 10 Weeks ]
  4. Proportion of subjects who require rescue therapy prior to Week 6 [ Time Frame: 2, 4, 5, 6 Weeks ]
  5. BPDAI (BP Disease Area Index) score [ Time Frame: 2, 4, 5, 6, 8, 10 Weeks ]
  6. Pruritus VAS (Visual Analogue Scale) score change from baseline [ Time Frame: 2, 4, 5, 6, 8, 10 Weeks ]
  7. DLQI (Dermatology Life Quality Index) score change from baseline [ Time Frame: 6, 10 Weeks ]
  8. Inflammation marker [ Time Frame: 6 Weeks ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   20 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Age 20 to 90 years old, inclusive, at enrollment.
  2. Diagnosis of bullous pemphigoid confirmed by histopathology and one of following assessments:

    1. Direct immunofluorescence (DIF)
    2. Indirect immunofluorescence (IIF)
    3. ELISA test (ELISA detection of immunoglobulin G (IgG) anti-BP180 autoantibodies in serum more than 9 U/mL).
  3. Localized or limited BP with the occurrence of <10 new blisters per day in the week prior to enrollment.
  4. Is male, or is female and meets all the following criteria:

    1. Not breastfeeding
    2. 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
  5. Is able to understand and sign the Informed Consent Form (ICF), communicate with the investigator, and understand and comply with protocol requirements.

Exclusion Criteria:

  1. 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.
  2. Use of oral steroids in the 2 weeks prior to enrollment at a dose greater than prednisolone equivalent dose (PED) of 10 mg/day.
  3. Use of topical steroids for more than 3 consecutive days in the 2 weeks prior to enrollment.
  4. 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.
  5. Use of systemic antibiotics in the 2 weeks prior to enrollment.
  6. Use of oral dapsone in the 2 weeks prior to enrollment.
  7. Treatment with intravenous immunoglobulin (IVIG) in the 8 weeks prior to enrollment.
  8. 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.
  9. Presence of active systemic infections.
  10. Any clinically significant medical condition or laboratory value that could potentially affect study participation and/or personal well-being, as judged by the investigator.
  11. History of allergy or hypersensitivity to any component of study medication or clobetasol.
  12. Has participated in a clinical study within 30 days prior to enrollment.
  13. 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.
  14. Is employed by the sponsor (i.e., is an employee, temporary contract worker, or designee responsible for the conduct of the study).

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03286582

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National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
TWi Biotechnology, Inc.
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Responsible Party: TWi Biotechnology, Inc. Identifier: NCT03286582    
Other Study ID Numbers: AC-203-BP-001
First Posted: September 18, 2017    Key Record Dates
Last Update Posted: September 19, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases
Anti-Inflammatory Agents
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs