A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03468322
• Date of registration: 08/03/2018
• Prospective Registration: Yes
• Primary sponsor: TWi Biotechnology, Inc.
• Public title: A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients
• Scientific title: A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa
• Date of first enrolment: October 20, 2018
• Target sample size: 9
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03468322
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Taiwan

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subject is at least 2 years of age.

2. Subject has a clinical diagnosis of EB.

3. Subject has a laboratory confirmed diagnosis of inherited EB based on electron
microscopy and/or immunofluorescence antigenic mapping.

4. Subject has two comparable areas with 1% - 5% BSA each. These two areas could be on
any body surface except the face, scalp, groin, palms and soles. Percentage BSA of the
designated areas within subject should be the same. Comparable areas are defined as
having similar lesion (i.e., blisters, erosions, erythema and crusts) history and
current lesion status by investigator's judgement on each area at Screening Visit
(Visit 1) and Day 1 (Visit 2).

5. 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 at Visit 1, and must practice and be
willing to continue to practice appropriate birth control during the entire
duration of the study.

6. Is able to read, understand, and sign the Informed Consent Form (ICF), answer the
study questionnaires, communicate with the investigator, and understand and comply
with protocol requirements, OR Informed consent received from subject's
parents/caregiver or legal guardian (when subject < 20 years).

Exclusion Criteria:

1. Subject has a current malignancy, or a history of treatment for a malignancy within
two years.

2. Systemic infections.

3. Subjects who are pregnant, lactating, or planning a pregnancy during the study.

4. History of allergy or hypersensitivity to any component of study medication.

5. Any other significant diseases, conditions, or laboratory values which, in the opinion
of the investigator, might make participation not in the subject's best interest or
confound the interpretation of study results.

6. Any prior use of approved or investigational biologic anti-inflammatory therapy within
6 months prior to screening, including but not limited to: anakinra, rilonacept,
canakinumab, etanercept, adalimumab, infliximab, rituximab, certolizumab, golimumab,
tocilizumab, bertilimumab, or abatacept.

7. Use of non-steroid immunosuppressants including but not limited to azathioprine,
mycophenolate, cyclophosphamide, chlorambucil, methotrexate, tacrolimus, or
cyclosporine in the 2 weeks prior to screening.

8. Has been treated with gentamicin within 90 days prior to screening (Note: products
containing gentamicin used on eyes are allowed).

9. Has been treated with minocycline, oxytetracycline, tetracycline or doxycycline within
7 days prior to screening.

10. Subjects has used any topical allantoin = 3% within 30 days prior to screening.

11. Has been treated systemic steroid within 30 days prior to screening.

12. Prior treatment with any investigational therapy within 30 days prior to screening.

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 sponsor (i.e., is an employee, temporary contract worker, or designee
responsible for the conduct of the study).



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Inherited Epidermolysis Bullosa

Intervention(s)

Drug: AC-203

Drug: Vehicle

Primary Outcome(s)

Percentage change in lesion surface area from baseline by treatment [Time Frame: 2, 4, 5, 6, 8, 12 Weeks]

Secondary Outcome(s)

hsCRP concentrations and changes from baseline [Time Frame: 8 Weeks]

Percentage change in blister number from baseline by treatment [Time Frame: 2, 4, 5, 6, 8, 12 Weeks]

Proportion of subjects with at least 40% reduction in blister number from baseline by treatment [Time Frame: 2, 4, 5, 6, 8, 12 Weeks]

Pruritus assessment scale changes from baseline by treatment [Time Frame: 2, 4, 5, 6, 8, 12 Week]

IL-1beta concentrations and changes from baseline [Time Frame: 8 Weeks]

Pain assessment scale changes from baseline by treatment [Time Frame: 2, 4, 5, 6, 8, 12 Weeks]

Secondary ID(s)

AC-203-EBS-005

Source(s) of Monetary Support

Please refer to primary and secondary sponsors