Trial Search Results

Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time. For certain phases of the study treatment assignment to the right and left foot will be randomized in a double blind fashion. Blood levels will test systemic absorption of sirolimus. Other safety and efficacy measures will be taken through the 39-week study duration. Funding Source - FDA OOPD

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

TransDerm, Inc.

Collaborator: Pachyonychia Congenita Project

Stanford Investigator(s):

Intervention(s):

  • Drug: 1% sirolimus cream (TD201 1%)

Phase:

Phase 1

Eligibility


Inclusion Criteria:

Subjects must:

   - Be capable of understanding the purpose and risks of the study and sign a written
   Informed Consent Form (ICF)

   - Be male or female ≥ 16 years of age at the time of the screening visit

   - Have a confirmed diagnosis of PC by genotyping (e.g., familial) and clinically
   correlated painful keratoderma.

   - Have roughly symmetrical calluses of similar severity on the plantar surface of both
   feet

   - Women of childbearing potential must have a negative serum pregnancy test

   - Subjects, whether male or female, with reproductive potential and who are sexually
   active must agree to use double-barrier contraception methods

Exclusion Criteria:

A Subject with any of the following criteria is not eligible for inclusion in this study:

   - Use of other investigational drugs within 30 days of the screening visit and/or has
   not recovered from any side effects of prior investigational drugs or procedure in the
   affected area (e.g., a biopsy)

   - Significant condition in the dermatologic treatment area such as trauma, or nonhealing
   chronic wound

   - Pregnant or nursing (lactating) female, or a positive serum pregnancy test

   - Active infection either systemic or local near the site of treatment requiring chronic
   or prolonged use of antimicrobial agents

   - Known immunodeficiency including: Hepatitis A; Hepatitis B; Hepatitis C; Human
   Immunodeficiency Virus (HIV)

Prior and Current Treatment

   - Unable to be discontinued from drugs known to either be inducers or inhibitors of
   CYP3A4/5 enzymes

   - Unable to be discontinued from drugs known to be P-glycoprotein inhibitors

Ages Eligible for Study

16 Years - N/A

Genders Eligible for Study

All

Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Joyce Teng, MD, PhD
650-723-6493
Not Recruiting