Clinical trial · Interventional
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
A Multicenter, Phase 2, Open-Label Study Evaluating The Safety And Efficacy of Sirolimus 3.9% Topical Gel (PTX-022) In The Treatment of Microcystic Lymphatic Malformations
NCT05050149CI-TRIAL-00084833completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Microcystic Lymphatic Malformation | Lymphangioma Circumscriptum | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| PTX-022 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Experimental: PTX-022
- description
- PTX-022 QTORIN
- interventionNames
- Drug: PTX-022
Primary outcomes (1)
- measure
- Incidence of Treatment-Emergent Adverse Events
- timeFrame
- 4 months
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 6 Years
Show eligibility criteria text
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.
References
Publications (1)
- DERIVEDTeng JMC, Hammill A, Martini J, Treat J. Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphat Res Biol. 2023 Apr;21(2):101-110. doi: 10.1089/lrb.2021.0103. Epub 2022 Jul 18. PMID 35852876