Phase 2
N=12
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
Microcystic Lymphatic Malformation
Bottom Line
View on ClinicalTrials.gov: NCT05050149 ↗Enrolled (actual)
12
Serious AEs
0.0%
Results posted
Jan 2025
Primary outcome: Primary: Incidence of Treatment-Emergent Adverse Events — 9; 2; 6; 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PTX-022 (Drug)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- All
- Sponsor
- Palvella Therapeutics, Inc.
- Primary completion
- Nov 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Treatment-Emergent Adverse Events |
9; 2; 6; 2; 1; 1 | — |
Summary
This study evaluates the safety and efficacy of PTX-022 (sirolimus) Topical Gel 3.9% w/w in the treatment of Microcystic Lymphatic Malformations. The participant will receive 3 months of PTX-022 treatment by the end of the study.
Eligibility Criteria
Inclusion Criteria
- Participants must be 6 years or older
- Diagnosed with Microcystic Lymphatic Malformations
- Able and willing to comply with all protocol-required 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
- Patient's 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.
Data sourced from ClinicalTrials.gov (NCT05050149). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.