PTX-022 (sirolimus) 3.9% topical gel for angiokeratomas.
A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Angiokeratomas (LOTU)
This trial will test whether PTX-022, a 3.9% sirolimus topical gel, is safe and helps people aged 6 and older who have angiokeratomas.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 15 (estimated) |
| Ages | 6 Years and up |
| Sex | All |
| Sponsor | Palvella Therapeutics, Inc. Industry-sponsored |
| Locations | 4 sites (Sherman Oaks, California and 3 other locations) |
| Trial ID | NCT07552077 on ClinicalTrials.gov |
What this trial studies
This phase 2, baseline-controlled interventional trial tests a 3.9% sirolimus topical gel (PTX-022) applied to angiokeratoma lesions. Participants aged 6 years and older with diagnosed angiokeratomas will apply the gel according to the protocol and attend scheduled in-person visits at one of three U.S. sites for monitoring. Outcomes compare lesion appearance and symptoms to each participant's baseline while tracking adverse events to characterize safety. The trial excludes patients with known sirolimus allergy or significant concurrent conditions that could affect participation or safety.
Who should consider this trial
Good fit: People aged 6 years or older with a confirmed diagnosis of angiokeratomas who can follow study procedures and give informed consent (or have a guardian consent) are the intended candidates.
Not a fit: Patients with a known allergy to sirolimus, significant concurrent medical conditions, or lesions that are deep/subcutaneous and unlikely to respond to topical therapy may not benefit from this treatment.
Why it matters
Potential benefit: If successful, the gel could shrink or fade angiokeratomas and reduce symptoms such as bleeding or irritation with a topical, noninvasive option.
How similar studies have performed: Topical sirolimus has shown benefit in other vascular and cutaneous lesions, but its use specifically for angiokeratomas is relatively novel and not yet well established.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Participants must be 6 years or older * Diagnosed with angiokeratoma * Able and willing to comply with all protocol-related activities * Willing and able to provide written informed consent Exclusion Criteria: * Any significant concurrent condition that could adversely affect participation * Any history of allergy or hypersensitivity to sirolimus, or sirolimus-like medications or to PTX-022 * Patients deemed by the investigator as unwilling or unable to remain compliant with all tests and procedures, including adherence to study drug administration and other protocol-required activities
Where this trial is running
Sherman Oaks, California and 3 other locations
- Unified Health — Sherman Oaks, California, United States (Not_yet_recruiting)
- Cleaver Medical Group — Dawsonville, Georgia, United States (Recruiting)
- Vividia Dermatology — Las Vegas, Nevada, United States (Not_yet_recruiting)
- Epiphany Dermatology — Southlake, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Emily Cook
- Email: emily.cook@palvellatx.com
- Phone: 267-738-6366
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.