Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations

Completed · Phase 2

Conditions studied: Microcystic Lymphatic Malformation

In brief

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.

Key facts

Study ID: NCT05050149
Run by: Palvella Therapeutics, Inc.
People needed: 12
Starts: 2022-01-13
Expected to finish: 2022-12-15
Last updated by the study team: 2025-01-17

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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.

Where it is running

Arkansas Children's Hospital — Little Rock, Arkansas, United States
Stanford University — Palo Alto, California, United States
Minnesota Clinical Study Center — Fridley, Minnesota, United States
Vascular Birthmark Institute — New York, New York, United States
Cincinnati Children's Hospital — Cincinnati, Ohio, United States
Oregon Health and Science University — Portland, Oregon, United States
Children's Hospital of Pennsylvania (CHOP) — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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