A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations
- Conditions
- Cutaneous Venous Malformations
- Interventions
- Registration Number
- NCT06653842
- Lead Sponsor
- Palvella Therapeutics, Inc.
- Brief Summary
A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX022 (sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 15
The participant must have a clinically confirmed superficial/ cutaneous (visible on the skin) venous malformation (cVM), referred to as the treatment area. The treatment area may contain a minority (<30%) lymphatic component (mixed venous / lymphatic malformation).
- The participant's treatment area is mainly in any wet mucosa or within the orbital rim. For clarification, no part of the venous malformation should be evaluated or treated if it in mucosa. External genital presentation is permitted.
- The participant has a known pervasive extension of the lesion into mucosa, bone or tissue (as determined by diagnostic or the investigator) that would impair the ability of the investigator to evaluate the cutaneous components of the lesion.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Single-arm, open-label QTORIN 3.9% rapamycin anhydrous gel -
- Primary Outcome Measures
Name Time Method Incidence of Treatment-Emergent Adverse Events Week 12
- Secondary Outcome Measures
Name Time Method Change in Investigator Global Assessment (cVM-IGA) From Baseline to Week 12 Change from baseline to Week 12 Likert scale used by clinicians to evaluate the overall change of the study designated venous malformation
Overall Patient Global Impression of Change (PGI-C) From Baseline to Week 12 Change from baseline to Week 12 Likert scale used by patients to evaluate the overall change of the study designated venous malformation
Trial Locations
- Locations (10)
Children's Hospital of Orange County
🇺🇸Irvine, California, United States
Colorado Children's Hospital
🇺🇸Aurora, Colorado, United States
John's Hopkins
🇺🇸Baltimore, Maryland, United States
University of North Carolina
🇺🇸Chapel Hill, North Carolina, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
University of Utah Health
🇺🇸Salt Lake City, Utah, United States
Stanford University
🇺🇸Palo Alto, California, United States
Minnesota Clinical Study Center
🇺🇸New Brighton, Minnesota, United States
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States