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Trial record 1 of 1 for:    AC-203 | Epidermolysis Bullosa
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A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients

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.
 
ClinicalTrials.gov Identifier: NCT03468322
Recruitment Status : Completed
First Posted : March 16, 2018
Last Update Posted : April 16, 2019
Sponsor:
Information provided by (Responsible Party):
TWi Biotechnology, Inc.

Tracking Information
First Submitted Date  ICMJE March 8, 2018
First Posted Date  ICMJE March 16, 2018
Last Update Posted Date April 16, 2019
Actual Study Start Date  ICMJE October 20, 2018
Actual Primary Completion Date April 9, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 16, 2019)
Percentage change in lesion surface area from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12 Weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: March 14, 2018)
Percentage change in lesion surface area from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12, 16 Weeks ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: January 16, 2019)
  • 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 ]
    100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)
  • Pain assessment scale changes from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12 Weeks ]
    100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)
  • IL-1beta concentrations and changes from baseline [ Time Frame: 8 Weeks ]
  • hsCRP concentrations and changes from baseline [ Time Frame: 8 Weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 14, 2018)
  • Percentage change in blister number from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12, 16 Weeks ]
  • Proportion of subjects with at least 40% reduction in blister number from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12, 16 Weeks ]
  • Pruritus assessment scale changes from baseline by treatment [ Time Frame: 2, 4, 5, 6, 8, 12, 16 Weeks ]
  • Inflammatory cytokine 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, 16 Weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients
Official Title  ICMJE A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa
Brief Summary

Inherited epidermolysis bullosa (EB) is a genetic skin disorder characterized by skin fragility and recurrent blister formation. More and more evidence has suggested that the skin lesions initially caused by genetic mutations may be further aggravated by inflammatory responses. Several reports showed successful alleviation of EB symptoms upon treatment with immunomodulatory therapies. Modulation of proinflammatory cytokine IL-1β has shown promising results in alleviating epidermolysis bullosa simplex (EBS), a major subtype of inherited EB, by downregulating IL-1β-mediated JNK/MAPK signaling pathway. This data further supports the potential of using cytokine modulators to treat EB.

AC-203, a topical formulation, can inhibit the production and activity of IL-1β, down-regulate IL-1β receptors, and increase IL1β-receptor antagonist (IL1-Ra) expression. In addition, AC-203 has been reported to inhibit anti-BP180 autoantibody-induced IL-6/IL-8 upregulation in cultured keratinocytes and LPS-induced IL-6 upregulation in cultured macrophages. Furthermore, AC-203 was also found to inhibit the formation of NLRP3 inflammasome, which plays essential roles in induction of caspase-1-dependent pyroptosis and release of inflammatory cytokines IL-1β and IL-18. These studies demonstrated the cytokine modulatory properties of AC-203 and pointed out the possible application of AC-203 in a variety of inflammatory diseases.

This study is designed to test the efficacy, safety, tolerability, and pharmacokinetics of AC-203 ointment (vs. placebo) in patients with inherited EB.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Intra-individual comparison
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Inherited Epidermolysis Bullosa
Intervention  ICMJE
  • Drug: AC-203
    The investigational product is formulated as 1% topical ointment
  • Drug: Vehicle
    Vehicle-only control study medication is the same formulation as investigational product without active ingredient
Study Arms  ICMJE
  • Experimental: AC-203 1% ointment
    AC-203 1% ointment, QD
    Intervention: Drug: AC-203
  • Placebo Comparator: Vehicle ointment
    Vehicle ointment, QD
    Intervention: Drug: Vehicle
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: January 16, 2019)
9
Original Estimated Enrollment  ICMJE
 (submitted: March 14, 2018)
8
Actual Study Completion Date  ICMJE April 9, 2019
Actual Primary Completion Date April 9, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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).
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Taiwan
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03468322
Other Study ID Numbers  ICMJE AC-203-EBS-005
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party TWi Biotechnology, Inc.
Study Sponsor  ICMJE TWi Biotechnology, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account TWi Biotechnology, Inc.
Verification Date April 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP