Cutaneous Venous Malformations
Conditions
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
Interventions
QTORIN 3.9% Rapamycin Anhydrous Gel
Sponsors
Palvella Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
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). \-
Exclusion criteria
* 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of Treatment-Emergent Adverse Events | Week 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
United States
Outcome results
None listed