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
Phase 3
Completed
- Conditions
- Pachyonychia Congenita
- Interventions
- Drug: Vehicle
- Registration Number
- NCT05180708
- Lead Sponsor
- Palvella Therapeutics, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 87
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Active QTORIN 3.9% rapamycin anhydrous gel - Vehicle Vehicle -
- Primary Outcome Measures
Name Time Method Patient Global Assessment of Activities Difficulty Scale 6 months
- Secondary Outcome Measures
Name Time Method PROMIS physical function 6 months Clinician global impression of severity (CGI-S) 6 months Pain at its worst as assessed by numerical rating scale 6 months Clinician global impression of change (CGI-C) 6 months Patient global impression of severity (PGI-S) 6 months Patient global impression of change (PGI-C) 6 months
Trial Locations
- Locations (8)
Oregon Health and Sciences University
🇺🇸Portland, Oregon, United States
Stanford University
🇺🇸Palo Alto, California, United States
Park Avenue Dermatology
🇺🇸Orange Park, Florida, United States
Minnesota Clinical Study Center
🇺🇸Fridley, Minnesota, United States
Royal London Hospital
🇬🇧London, Whitechapel, United Kingdom
DermResearch, Inc.
🇺🇸Austin, Texas, United States
University of Utah
🇺🇸Murray, Utah, United States
Yale University
🇺🇸New Haven, Connecticut, United States