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Inherited Epidermolysis BullosaPhase 2NCT03468322

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

A Phase 2 study testing AC-203 in people with Inherited Epidermolysis Bullosa, run by TWi Biotechnology, Inc. at 2 sites in Taiwan.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Inherited Epidermolysis Bullosa
What is being tested?
AC-203
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
9 participants
Where
2 sitesTaiwan
Study dates
20 Oct 2018to 9 Apr 2019
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Subject is at least 2 years of age.
  • Subject has a clinical diagnosis of EB.
  • Subject has a laboratory confirmed diagnosis of inherited EB based on electron microscopy and/or immunofluorescence antigenic mapping.
  • 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).
  • 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 at Visit 1, and must practice and be willing to continue to practice appropriate birth control during the entire duration of the study.
  • 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).

Who cannot join

  • Subject has a current malignancy, or a history of treatment for a malignancy within two years.
  • Systemic infections.
  • Subjects who are pregnant, lactating, or planning a pregnancy during the study.
  • History of allergy or hypersensitivity to any component of study medication.
  • 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.
  • 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.
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  • 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.
  • Has been treated with gentamicin within 90 days prior to screening (Note: products containing gentamicin used on eyes are allowed).
  • Has been treated with minocycline, oxytetracycline, tetracycline or doxycycline within 7 days prior to screening.
  • Subjects has used any topical allantoin ≥ 3% within 30 days prior to screening.
  • Has been treated systemic steroid within 30 days prior to screening.
  • Prior treatment with any investigational therapy within 30 days prior to screening.
  • 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 sponsor (i.e., is an employee, temporary contract worker, or designee responsible for the conduct of the study).

Trial locations and contacts

2 sites in 1 country.

Taiwan2 sites

  • Mackay Memorial Hospital

    Hsinchu

  • National Cheng Kung University Hospital

    Tainan

Other trials in Taiwan

About this study

Official title: A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa

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.

Being tested
AC-203, Vehicle

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AC-203Drug

    The investigational product is formulated as 1% topical ointment

  • VehicleDrug

    Vehicle-only control study medication is the same formulation as investigational product without active ingredient

Study groups in NCT03468322
Study groupRoleWhat they receive
AC-203 1% ointmentExperimentalAC-203 1% ointment, QD
Receives: Drug: AC-203
Vehicle ointmentPlacebo comparatorVehicle ointment, QD
Receives: Drug: Vehicle

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Percentage change in lesion surface area from baseline by treatment

    Measured: 2, 4, 5, 6, 8, 12 Weeks

Secondary outcomes (6)

  1. Percentage change in blister number from baseline by treatment

    Measured: 2, 4, 5, 6, 8, 12 Weeks

  2. Proportion of subjects with at least 40% reduction in blister number from baseline by treatment

    Measured: 2, 4, 5, 6, 8, 12 Weeks

  3. Pruritus assessment scale changes from baseline by treatment

    100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)

    Measured: 2, 4, 5, 6, 8, 12 Week

  4. Pain assessment scale changes from baseline by treatment

    100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)

    Measured: 2, 4, 5, 6, 8, 12 Weeks

  5. IL-1beta concentrations and changes from baseline

    Measured: 8 Weeks

  6. hsCRP concentrations and changes from baseline

    Measured: 8 Weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2018
  2. Study start20 Oct 2018
  3. Primary completion9 Apr 2019
  4. Study completion9 Apr 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Intra-individual comparison
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
9 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03468322
Sponsor's protocol ID
AC-203-EBS-005
Status last verified
Apr 2019
First submitted
8 Mar 2018
Last updated
16 Apr 2019
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing AC-203 in people with Inherited Epidermolysis Bullosa, run by TWi Biotechnology, Inc. at 2 sites in Taiwan.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 8 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Percentage change in lesion surface area from baseline by treatment (time frame: 2, 4, 5, 6, 8, 12 Weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03468322.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Apr 2019.

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