Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
- Registration Number
- NCT03920228
- Lead Sponsor
- Palvella Therapeutics, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 73
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Open label period PTX-022 - Randomized period - Dosing A Placebo - Randomized period - Dosing B PTX-022 - Randomized period - Dosing B Placebo - Randomized period - Dosing C PTX-022 -
- Primary Outcome Measures
Name Time Method Patient Global Assessment of Activities Scale 6 months
- Secondary Outcome Measures
Name Time Method Clinician Global Impression of Change Scale 6 months Pain at its worst as assessed by numerical rating scale 6 months Number of steps taken as assessed by activity monitor 6 months
Trial Locations
- Locations (9)
International Dermatology Research
🇺🇸Miami, Florida, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Arizona Research Center
🇺🇸Phoenix, Arizona, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
University of Utah
🇺🇸Murray, Utah, United States
Stanford University
🇺🇸Palo Alto, California, United States
Minnesota Clinical Study Center
🇺🇸Fridley, Minnesota, United States
Paddington Testing Co.
🇺🇸Philadelphia, Pennsylvania, United States
Yale University
🇺🇸New Haven, Connecticut, United States