Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Condition(s) targeted: Pachyonychia Congenita

Intervention: 1% sirolimus cream (TD201 1%) (Drug); placebo cream (vehicle control) (Drug)

Phase: Phase 1/Phase 2

Status: Active, not recruiting

Sponsored by: TransDerm, Inc.

Official(s) and/or principal investigator(s):
Roger L Kaspar, PhD, Study Chair, Affiliation: TransDerm, Inc.

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

Official title: Phase 1b Clinical Trial Using Topical Sirolimus for the Treatment of Pachyonychia Congenita

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome: Evaluation of systemic absorption through measurement of serum sirolimus trough levels

Secondary outcome:

Weekly assessments recorded in the PC Quality of Life Index

Daily assessments recording in the PC Measurement Diary

Minimum age: 16 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

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

Stanford University, Stanford, California 94305, United States

Starting date: January 2014
Last updated: June 17, 2015