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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
73
Arizona Research Center
Phoenix, Arizona, United States
Stanford University
Palo Alto, California, United States
Yale University
New Haven, Connecticut, United States
International Dermatology Research
Patient Global Assessment of Activities Scale
Time frame: 6 months
Clinician Global Impression of Change Scale
Time frame: 6 months
Pain at its worst as assessed by numerical rating scale
Time frame: 6 months
Number of steps taken as assessed by activity monitor
Time frame: 6 months
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Miami, Florida, United States
Northwestern University
Chicago, Illinois, United States
Minnesota Clinical Study Center
Fridley, Minnesota, United States
Oregon Health and Science University
Portland, Oregon, United States
Paddington Testing Co.
Philadelphia, Pennsylvania, United States
University of Utah
Murray, Utah, United States