A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita

• QTORIN 3.9% rapamycin anhydrous gel (Drug): QTORIN 3.9% rapamycin anhydrous gel
• Vehicle (Drug): Matching vehicle (QTORIN anyhydrous gel)

Primary outcomes

• Patient Global Assessment of Activities Difficulty Scale [6 months]

Secondary outcomes

• Pain at its worst as assessed by numerical rating scale [6 months]
• PROMIS physical function [6 months]
• Patient global impression of severity (PGI-S) [6 months]
• Patient global impression of change (PGI-C) [6 months]
• Clinician global impression of severity (CGI-S) [6 months]
• Clinician global impression of change (CGI-C) [6 months]

Sex: All, ages: 18 Years to any

Key Inclusion Criteria:

* Adult patients, 18 years or older (16 years or older in the UK)
* Diagnosed with Pachyonychia Congenita (PC), genetically confirmed

Key Exclusion Criteria:

* Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications.
* Any significant concurrent condition (including conditions involving or inferior to the ankle) that could adversely affect the patient's participation and/or the assessment of the safety and efficacy in the study.
* Participation in any other interventional clinical research trial within 30 days prior to the screening visit or during the patient's participation in this study

• Amy Paller, MD, Study chair, Northwestern University
• Joyce Teng, MD, Principal investigator, Stanford University
• David Hansen, MD, Principal investigator, University of Utah