Topical sirolimus (PTX-022) for microcystic lymphatic malformations
SELVA: A Multicenter, Phase 3 Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
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 ID | NIH-11196066 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: 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.
Not a fit: 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.
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 performed: 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
Wayne, United States
- Palvella Therapeutics, INC. — Wayne, United States (Active)
Researchers
- Principal investigator: Martini, Jeff — Palvella Therapeutics, INC.
- Study coordinator: Martini, Jeff
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 below.