Topical sirolimus (PTX-022) for microcystic lymphatic malformations
Official title: SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Funded at Palvella Therapeutics, INC. · NIH-11196066 · Data updated 2026-11-12
This project offers a topical sirolimus gel (PTX-022) for people with microcystic lymphatic malformations to try to reduce leaking, infections, and other skin symptoms while checking safety.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Palvella Therapeutics, INC. NIH-funded |
| Lab location | 1 site (Wayne, United States) |
| Project start | September 20, 2024 |
| Project end | August 31, 2028 |
| Funding year | 2025 |
| Award (this year) | $648,834 |
| NIH project number | 5R01FD008196-02 |
| Project ID | NIH-11196066 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are people diagnosed with microcystic lymphatic malformations who have symptomatic skin lesions such as lymphorrhea or recurrent cellulitis and who can attend clinic visits.
✕Less likely to help
People without microcystic LM, those whose disease is primarily deep or macrocystic, or patients with contraindications to sirolimus (for example allergy or certain immunosuppressive conditions) may not benefit.
What this research studies
You would receive a topical sirolimus gel applied to affected skin areas as part of a multicenter, phase 3, baseline-controlled protocol. The study compares your symptoms and complications before and after treatment to measure benefit and records side effects and safety data. Visits at study centers will collect clinical exams, photographs, and possibly patient-reported symptom measures. The goal is to determine whether a topical formulation can improve day-to-day symptoms like lymphorrhea and reduce episodes of cellulitis with fewer systemic side effects than oral treatment.
Why it matters
Potential benefit: If successful, this could become the first FDA-approved topical treatment to reduce leaking, infections, and skin problems from microcystic lymphatic malformations.
How similar studies have gone: Oral sirolimus (systemic rapamycin) has shown preliminary benefit for microcystic LM, and early topical/compassionate-use reports suggest promise, but a phase 3 trial like this is needed for definitive evidence and approval.
Where this research is happening
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Palvella Therapeutics, INC.
Wayne, United States Active
Researchers
- Principal investigator: Martini, Jeff — Palvella Therapeutics, INC.
- Study coordinator: Martini, Jeff
Related research
Other NIH-funded projects on the same topics.
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PTX-022 ointment gel for rare microcystic lymphatic malformations (LM)
A new topical gel (PTX-022) is being tested to help people with a rare, lifelong skin and lymphatic condition called microcystic LM.
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Evaluating a new treatment for microcystic lymphatic malformations
This study is testing a new gel called PTX-022 to see if it can safely help people with microcystic lymphatic malformations, a rare condition that affects the lymphatic system, and it's looking for participants to see how well it works and if there are any side effects.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.