Cutaneous Venous MalformationsPhase 2TOIVANCT06653842
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
A Phase 2 study testing QTORIN 3.9% rapamycin anhydrous gel in people with Cutaneous Venous Malformations, run by Palvella Therapeutics, Inc. at 10 sites in United States.
Trial 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 Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cutaneous Venous Malformations
- What is being tested?
- QTORIN 3.9% rapamycin anhydrous gel
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 10 sitesUnited States
- Study dates
- 20 Dec 2024to Mar 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- 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 locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Colorado Children's Hospital
Aurora, Colorado, 80045
Johns Hopkins
Baltimore, Maryland, 21287
University of North Carolina
Chapel Hill, North Carolina, 27516
Cleveland Clinic
Cleveland, Ohio, 44195
Children's Hospital of Orange County
Irvine, California, 92612
Minnesota Clinical Study Center
New Brighton, Minnesota, 55112
Stanford University
Palo Alto, California, 94305
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah Health
Salt Lake City, Utah, 84113
About this study
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
- Conditions
- Cutaneous Venous Malformations
- Being tested
- QTORIN 3.9% rapamycin anhydrous gel
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
QTORIN 3.9% rapamycin anhydrous gelDrug
QTORIN 3.9% Rapamycin Anhydrous Gel
| Study group | Role | What they receive |
|---|---|---|
| Single-arm, open-label | Experimental | Not described Receives: Drug: QTORIN 3.9% rapamycin anhydrous gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events
Measured: Week 12
Secondary outcomes (2)
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
Measured: 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
Measured: Change from baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2024
- Study start20 Dec 2024
- Primary completion21 Nov 2025
- Study completionMar 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cutaneous Venous MalformationsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Palvella Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06653842
- Sponsor's protocol ID
- PALV-10
- Acronym
- TOIVA
- Status last verified
- Mar 2026
- First submitted
- 17 Oct 2024
- Last updated
- 27 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06653842
- Acronym / sponsor ID
- TOIVA / PALV-10
- Conditions
- Cutaneous Venous Malformations
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Palvella Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Dec 2024 (actual)
- Primary completion
- 21 Nov 2025 (actual)
- Study completion
- Mar 2026 (estimated)
- First posted
- 22 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single-arm, open-label | Experimental | Drug: QTORIN 3.9% rapamycin anhydrous gel |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| QTORIN 3.9% rapamycin anhydrous gelQTORIN 3.9% Rapamycin Anhydrous Gel | Drug | Single-arm, open-label |
Primary outcome
Incidence of Treatment-Emergent Adverse Events
Time frame: Week 12
2 secondary outcomes
Change in Investigator Global Assessment (cVM-IGA) From Baseline to Week 12
Time frame: Change from baseline to Week 12
Overall Patient Global Impression of Change (PGI-C) From Baseline to Week 12
Time frame: Change from baseline to Week 12
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2026.
- Related InfoLink provided by the sponsor.
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing QTORIN 3.9% rapamycin anhydrous gel in people with Cutaneous Venous Malformations, run by Palvella Therapeutics, Inc. at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (time frame: Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2026.