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)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Cutaneous Venous Malformations
• Intervention / Treatment: Drug: QTORIN 3.9% rapamycin anhydrous gel

• Study Type: Interventional
• Enrollment (Estimated): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92612
      • Children's Hospital of Orange County
    • Palo Alto, California, United States, 94305
      • Stanford University
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Colorado Children's Hospital
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Johns Hopkins
  • Minnesota
    • New Brighton, Minnesota, United States, 55112
      • Minnesota Clinical Study Center
    • Rochester, Minnesota, United States, 55905
      • Mayo Clinic
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27516
      • University of North Carolina
  • Ohio
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Children's Hospital of Philadelphia
  • Utah
    • Salt Lake City, Utah, United States, 84113
      • University of Utah Health

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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).

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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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Single-arm, open-label	Drug: QTORIN 3.9% rapamycin anhydrous gel; QTORIN 3.9% Rapamycin Anhydrous Gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Incidence of Treatment-Emergent Adverse Events	Week 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Investigator Global Assessment (cVM-IGA) From Baseline to Week 12	Likert scale used by clinicians to evaluate the overall change of the study designated venous malformation	Change from baseline to Week 12
Overall Patient Global Impression of Change (PGI-C) From Baseline to Week 12	Likert scale used by patients to evaluate the overall change of the study designated venous malformation	Change from baseline to Week 12

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cutaneous Venous Malformation multicomponent static scale	Sum of the two individual component scores for height/swelling and appearance.	12 weeks

Collaborators and Investigators

• Sponsor: Palvella Therapeutics, Inc.

Publications and helpful links

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): December 20, 2024
• Primary Completion (Actual): November 21, 2025
• Study Completion (Estimated): March 1, 2026

Study Registration Dates

• First Submitted: October 17, 2024
• First Submitted That Met QC Criteria: October 21, 2024
• First Posted (Actual): October 22, 2024

Study Record Updates

• Last Update Posted (Actual): March 27, 2026
• Last Update Submitted That Met QC Criteria: March 26, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Congenital Abnormalities; Anti-Bacterial Agents; Anti-Infective Agents; Antibiotics, Antineoplastic; Antineoplastic Agents; Antifungal Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Sirolimus
• Other Study ID Numbers: PALV-10

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes