EGFR Inhibition for Skin Disorders
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores whether a low dose of erlotinib, an EGFR inhibitor, can help with keratinopathies, which cause thick, scaly skin. Erlotinib is already used in cancer treatment, but researchers believe it might also reduce skin scaling and pain in these conditions. The trial aims to understand erlotinib's effects on skin cell growth and the immune response. Suitable participants have a confirmed diagnosis of a keratinopathy, such as Epidermolytic ichthyosis or Pachyonychia congenita, and experience moderate symptoms. As a Phase 1/Phase 2 trial, it focuses on understanding the treatment's effects in people and measuring its initial effectiveness.
Will I have to stop taking my current medications?
The trial requires that you stop taking oral retinoids or oral steroids at least 4 weeks before starting the study drug. You must also avoid any investigational drugs or biologics within 4 weeks or 5 half-lives before starting the trial. Otherwise, stable medications that you've been on for at least a month before the observation period can be continued.
Is there any evidence suggesting that this treatment is likely to be safe for humans?
In a previous study, researchers found that erlotinib, a cancer treatment drug, often causes side effects like skin rashes and diarrhea, especially at higher doses. However, these side effects occur less frequently with lower doses.
Erlotinib also serves as the main treatment for Olmsted syndrome, a rare skin disorder. Reports have shown it helps manage symptoms in both children and adults with this condition. While effective, some individuals may still experience common side effects, such as dry skin and itching, which are generally manageable and expected as part of the treatment.
For those considering joining a trial, it's important to know that while side effects can occur, they are usually mild and less frequent at lower doses.12345Why do researchers think this study treatment might be promising?
Erlotinib is unique because it targets the epidermal growth factor receptor (EGFR), a protein that plays a key role in the growth and survival of skin cells. Unlike standard treatments for skin disorders, which often involve topical steroids or systemic immunosuppressants, erlotinib directly interferes with the signaling pathways that can cause abnormal skin cell proliferation. Researchers are excited about erlotinib because it offers a targeted approach that could potentially result in fewer side effects and more effective management of certain skin conditions, especially those driven by EGFR activity.
What evidence suggests that erlotinib might be an effective treatment for keratinopathies?
Research has shown that erlotinib, a type of medication, has potential in treating certain skin disorders. Participants in this trial will receive erlotinib, which has already proven effective for Olmsted syndrome, a rare skin condition, and is considered the best treatment option. Erlotinib targets a specific pathway in the body that affects skin cell growth and development. This pathway plays a crucial role in conditions impacting the skin's structure. Early findings suggest that a low dose of erlotinib can improve skin scaling and thickening and may help manage pain, especially in conditions affecting movement. Increasing evidence indicates that erlotinib could also help with other similar skin disorders.678910
Who Is on the Research Team?
Amy Paller
Principal Investigator
Northwestern University
Are You a Good Fit for This Trial?
This trial is for individuals with certain skin conditions like Epidermolytic Hyperkeratosis and Palmoplantar Keratoderma. Participants should have symptoms such as thickened skin or malformed nails. The study is open to those who can tolerate oral medication and are not currently using other treatments that might interfere with the study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Observation
Natural history of keratinopathies and baseline scores determined
Dose Escalation
Participants receive escalating doses of erlotinib (50 mg, 75 mg, 100 mg) to assess safety and efficacy
Open-label Extension
Participants continue dosing for an additional 6 months based on tolerance and effect
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- Erlotinib
Erlotinib is already approved in European Union, United States, Canada for the following indications:
- Non-small cell lung cancer
- Pancreatic cancer
- Non-small cell lung cancer
- Pancreatic cancer
- Non-small cell lung cancer
- Pancreatic cancer
Find a Clinic Near You
Who Is Running the Clinical Trial?
Northwestern University
Lead Sponsor
Yale University
Collaborator