A Safety and Tolerability Study of Topical PAT-001 in Congenital Ichthyosis
Condition: Congenital Ichthyosis
MeSH terms: Ichthyosis; Ichthyosis, Lamellar; Ichthyosiform Erythroderma, Congenital
Intervention: PAT-001, 0.1%; PAT-001, 0.2%; Vehicle for PAT-001 0.1%; Vehicle for PAT-001 0.2%
Intervention description: PAT-001 is a topical ointment. PAT-001, 0.1% contains 0.1% of active drug.; PAT-001 is a topical ointment. PAT-001, 0.2% contains 0.2% of active drug.; Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.1%.; Vehicle topical ointment contains 0.0% of active drug and is color matched to the active test article, PAT-001 0.2%.
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 19.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No
Keywords: X-linked, lamellar

Summary:

      Congenital ichthyosis (CI) is a large, heterogeneous family of inherited skin disorders of
      cornification resulting from an abnormality of skin keratinization, such as scaling and
      thickening of the skin. Treatment options include keratolytic agents, which can abruptly lead
      to extensive shedding or peeling of scales. PAT-001 primarily acts as a keratolytic agent;
      thus, making it a potential drug candidate for the treatment of skin disorders associated
      with hyperkeratinization, such as CI. The current study intends to evaluate the safety and
      tolerability of PAT-001 in patients with CI of either the Lamellar or X-Linked subtypes.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Patients of either sex aged 12 years or older.

          -  Females of childbearing potential should use appropriate contraception. Women of
             childbearing potential must have a negative pregnancy test at screening and baseline
             visits.

          -  Patient and legal representative(s), if applicable, has provided written informed
             consent.

          -  Patient has congenital ichthyosis of either lamellar or X-Linked subtype.

          -  Patient has two contralateral comparable Treatment Areas (e.g., each arm is affected
             and treatments areas can be applied equally).

          -  Patient is, except for their ichthyosis, in good general health.

        Exclusion Criteria:

          -  Patient is pregnant or breast feeding, or is planning to become pregnant during the
             study.

          -  Patient has inflammatory skin disease unrelated to ichthyosis.

          -  Patient is currently using concomitant retinoid therapy, within two weeks (topical) or
             12 weeks (oral) of Visit 2/Baseline.

          -  Patient is currently taking concomitant immunosuppressive drugs, including systemic
             corticosteroids, within two weeks of Visit 2/Baseline.

          -  Patient is currently enrolled in an investigational drug or device study.

          -  Patient has used an investigational drug or investigational device treatment within 30
             days prior to Visit 2/Baseline.

          -  Patient is unable to communicate or cooperate with the investigator due to language
             problems, impaired cerebral function, or physical limitations.

          -  Patient is known to be noncompliant or is unlikely to comply with the requirements of
             the study protocol (e.g., due to alcoholism, drug dependency, mental incapacity) in
             the opinion of the investigator.