This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
87
QTORIN 3.9% rapamycin anhydrous gel
Matching vehicle (QTORIN anyhydrous gel)
Stanford University
Palo Alto, California, United States
Yale University
New Haven, Connecticut, United States
Park Avenue Dermatology
Orange Park, Florida, United States
Minnesota Clinical Study Center
Fridley, Minnesota, United States
Patient Global Assessment of Activities Difficulty Scale
Time frame: 6 months
Pain at its worst as assessed by numerical rating scale
Time frame: 6 months
PROMIS physical function
Time frame: 6 months
Patient global impression of severity (PGI-S)
Time frame: 6 months
Patient global impression of change (PGI-C)
Time frame: 6 months
Clinician global impression of severity (CGI-S)
Time frame: 6 months
Clinician global impression of change (CGI-C)
Time frame: 6 months
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Oregon Health and Sciences University
Portland, Oregon, United States
DermResearch, Inc.
Austin, Texas, United States
University of Utah
Murray, Utah, United States
Royal London Hospital
London, Whitechapel, United Kingdom