VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study. The purpose of the OLE study is to investigate long term exposure to investigational PTX-022 and evaluate safety and efficacy data. A sub-study is included to evaluate safety and efficacy of patients with genotyped keratin mutations of KRT6C and KRT17 not previously treated with investigational PTX-022.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Safety and Efficacy of PTX-022 in the Treatment of Adults with Pachyonychia Congenita
Arizona Research Center
Phoenix, Arizona, United States
Stanford University
Palo Alto, California, United States
Minnesota Clinical Study Center
Fridley, Minnesota, United States
University of Utah
Murray, Utah, United States
Incidence of Treatment-Emergent Adverse Events
Time frame: 18 Months
Changes in vital sign measurements from baseline
Time frame: 18 months
Changes in weight measurements from baseline
Time frame: 18 months
Changes in clinical laboratory measurements from baseline
Time frame: 18 months
Patient Global Assessment of Activities Scale
Time frame: 18 Months
Pain at its worst as assessed by NRS
Time frame: 18 months
PROMIS Pain Interference Short 6A as assessed by likert scale
Time frame: 18 months
PROMIS Physical Function as assessed by likert scale
Time frame: 18 months
Patient Global Impression of Severity as assessed by likert scale
Time frame: 18 months
Clinician Global Impression of Severity as assessed by likert scale
Time frame: 18 months
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