©2025 Stanford Medicine
Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
Not Recruiting
Trial ID: NCT03920228
Purpose
This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.
This study includes four-parts, and if a participant completes all parts, the participant will have received at least 3-months of PTX-022 treatment.
Official Title
A Multicenter, Four-Part, Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Adults With Moderate to Severe Pachyonychia Congenita
Stanford Investigator(s)
Joyce Teng, MD, PhD
Professor of Dermatology and, by courtesy, of Pediatrics
Eligibility
Inclusion Criteria:
* Adult patients, 18 years or older
* Diagnosed Pachyonychia Congenita (PC), genetically confirmed
* Moderate to Severe PC
* Able and willing to comply with all protocol-required activities
* Willing and able to provide written informed consent
Exclusion Criteria:
* Any significant concurrent condition (including involving the inferior to the ankle) that could adversely affect participation.
* Any intentional changes in the patient's daily activities (associated with standing, walking and keeping balance), not resulting from an improvement in the patient's condition due to treatment.
* Patient's deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities.
Intervention(s):
drug: PTX-022
drug: Placebo
Not Recruiting
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Karima
650-723-0636