NCT06653842

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

Trial Health

55
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
15

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2024

Shorter than P25 for phase_2

Geographic Reach
1 country

10 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 17, 2024

Completed
5 days until next milestone

First Posted

Study publicly available on registry

October 22, 2024

Completed
2 months until next milestone

Study Start

First participant enrolled

December 20, 2024

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 21, 2025

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2026

Completed
Last Updated

March 27, 2026

Status Verified

March 1, 2026

Enrollment Period

11 months

First QC Date

October 17, 2024

Last Update Submit

March 26, 2026

Conditions

Outcome Measures

Primary Outcomes (1)

  • Incidence of Treatment-Emergent Adverse Events

    Week 12

Secondary Outcomes (2)

  • Change in Investigator Global Assessment (cVM-IGA) From Baseline to Week 12

    Change from baseline to Week 12

  • Overall Patient Global Impression of Change (PGI-C) From Baseline to Week 12

    Change from baseline to Week 12

Study Arms (1)

Single-arm, open-label

EXPERIMENTAL
Drug: QTORIN 3.9% rapamycin anhydrous gel

Interventions

QTORIN 3.9% Rapamycin Anhydrous Gel

Single-arm, open-label

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

Children's Hospital of Orange County

Irvine, California, 92612, United States

Location

Stanford University

Palo Alto, California, 94305, United States

Location

Colorado Children's Hospital

Aurora, Colorado, 80045, United States

Location

Johns Hopkins

Baltimore, Maryland, 21287, United States

Location

Minnesota Clinical Study Center

New Brighton, Minnesota, 55112, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27516, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

University of Utah Health

Salt Lake City, Utah, 84113, United States

Location

Related Links

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 17, 2024

First Posted

October 22, 2024

Study Start

December 20, 2024

Primary Completion

November 21, 2025

Study Completion

March 1, 2026

Last Updated

March 27, 2026

Record last verified: 2026-03

Locations