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 (TOIVA)

Palvella Therapeutics

Status and phase: Enrolling; Phase 2

Conditions: Cutaneous Venous Malformations

Treatments: Drug: QTORIN 3.9% rapamycin anhydrous gel

Study type: Interventional

Funder types: Industry

Identifiers

NCT06653842

PALV-10

Details and patient eligibility

About

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

Enrollment: 15 estimated patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Single-arm, open-label

Experimental group

Treatment:

Drug: QTORIN 3.9% rapamycin anhydrous gel