Status:

NOT_YET_RECRUITING

Epidermal Growth Factor Receptor Inhibition for Keratinopathies

Lead Sponsor:

Northwestern University

Collaborating Sponsors:

Yale University

Conditions:

Epidermolytic Ichthyosis

Palmoplantar Keratoderma

Eligibility:

All Genders

18+ years

Phase:

PHASE1

PHASE2

Brief Summary

Epidermal growth factor receptor (EGFR) signaling plays a key role in regulating epidermal cell proliferation, survival, and differentiation. Keratins form a scaffold with epidermal desmosomes that in...

Detailed Description

We will conduct the first interventional multi-site Phase 1/2a trial of low-dose erlotinib to test its safety and efficacy as systemic treatment of moderate-to-severe keratinopathies. The trial will ...

Eligibility Criteria

Inclusion

  • The participant must be at least 18 years of age.
  • The participant must have a clinically and genetically confirmed diagnosis of a keratinopathy (Epidermolytic ichthyosis/EI: KRT 1, 2, or 10 pathogenic variant; Epidermolytic palmoplantar keratoderma/PPK: KRT 9; Pachyonychia congenita/PC form of PPK: KRT 6a, 6b, 6c, 16, 17). A blood or saliva sample will be collected for genetic confirmation if the underlying genetic change has not been confirmed by a CLIA-approved laboratory.
  • The participant must have an investigator scored IGA at screening and baseline of at least 2 (moderate) at affected sites.
  • The participant must abstain from use of any investigational drug or biologic within 4 weeks or 5 half-lives if longer, oral retinoid or oral steroid for at least 4 weeks, and topical medications (prescription or over-the-counter) in the week before the 8-week observation period. Otherwise stable medications (at least a month before the observation period and continued) will be allowed.
  • The participant agrees to use standard of care with respect to bathing and exfoliation/foot care, except in the week before the visits. Moisturizers/ emollients are allowed throughout the treatment period, but use must be consistent and withheld to the designated area of sampling on the arms (EI) or plantar foot (PC/PPK) for 48 hours prior to visits when skin samples are taken.
  • The participant is willing to have blood collected for safety and biopsies for investigation of mechanism.
  • Participant is in good general health and free of any known disease state or physical condition which, in the investigator's opinion, might impair the evaluation of the EI or PC/PPK.
  • Participant is able and willing to follow all study instructions

Exclusion

  • The participant has EI or PC/PPK lesions on the assessment area that are infected or require therapy to treat the infection within 7 days of enrollment.
  • The participant has had major surgery during the past 4 weeks.
  • Participants have received an investigational drug or biologic within 4 weeks or 5 half-lives, whichever is longer, prior to starting treatment with and during treatment with erlotinib.
  • Participants must be off oral retinoid or oral steroid for at least 4 weeks before drug initiation.
  • Participants who require medications and OTC supplements that inhibit/ induce CYP3A4 activity to control concurrent medical conditions.
  • Participants with known hypersensitivity to any of the ingredients in the study medication formulation.
  • Participants previously treated for invasive cancer within the past 5 years unless the Investigator concludes history of cancer is not confounding to safety.
  • Participants who are pregnant, breastfeeding or planning to become pregnant during the study or within a month after the study ends.
  • Participants of childbearing potential who are unwilling or unable to comply with contraception measures.
  • The participant has any condition or situation which, in the Investigator's opinion, may put the subject at significant risk, could confound the study results, or could interfere significantly with the subject's participation in the study.
  • Participants deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, adherence to the study drug administration regimen and other protocol-required activities.

Key Trial Info

Start Date :

December 1 2026

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

June 30 2030

Estimated Enrollment :

44 Patients enrolled

Trial Details

Trial ID

NCT06545695

Start Date

December 1 2026

End Date

June 30 2030

Last Update

August 15 2025

Active Locations (1)

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1

Department of Dermatology, Northwestern University Feinberg School of Medicine

Chicago, Illinois, United States, 60611

Epidermal Growth Factor Receptor Inhibition for Keratinopathies | DecenTrialz