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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03286582
• Date of registration: 14/09/2017
• Prospective Registration: No
• Primary sponsor: TWi Biotechnology, Inc.
• Public title: A Proof-of-Concept Study of Topical AC-203 in Patients With Bullous Pemphigoid
• Scientific title: A Randomized, Open-Label, Controlled Trial of Topical AC-203 in Subjects With Bullous Pemphigoid
• Date of first enrolment: September 5, 2017
• Target sample size: 10
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03286582
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Taiwan

Key inclusion & exclusion criteria

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 immuno?uorescence (DIF)

2. Indirect immuno?uorescence (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).



Age minimum: 20 Years
Age maximum: 90 Years
Gender: All

Health Condition(s) or Problem(s) studied

Bullous Pemphigoid

Intervention(s)

Drug: Clobetasol 0.05% Topical Ointment

Drug: AC-203 1% Topical Ointment

Primary Outcome(s)

Incidence of adverse events during the treatment period [Time Frame: 10 Weeks]

Secondary Outcome(s)

Time to disease control [Time Frame: 2, 4, 5, 6, 8, 10 Weeks]

New blister count [Time Frame: 2, 4, 5, 6, 8, 10 Weeks]

BPDAI (BP Disease Area Index) score [Time Frame: 2, 4, 5, 6, 8, 10 Weeks]

Pruritus VAS (Visual Analogue Scale) score change from baseline [Time Frame: 2, 4, 5, 6, 8, 10 Weeks]

Proportion of subjects who require rescue therapy prior to Week 6 [Time Frame: 2, 4, 5, 6 Weeks]

Inflammation marker [Time Frame: 6 Weeks]

Proportion achieving disease control (no new blisters within prior week) [Time Frame: 2, 4, 5, 6, 8, 10 Weeks]

DLQI (Dermatology Life Quality Index) score change from baseline [Time Frame: 6, 10 Weeks]

Secondary ID(s)

AC-203-BP-001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors