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

Phase 2

Completed

• Conditions: Microcystic Lymphatic Malformation
• Interventions: Drug: PTX-022

• Registration Number: NCT05050149
• Lead Sponsor: Palvella Therapeutics, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-09-08
• Study First Submitted QC: 2021-09-17
• Study First Posted: 2021-09-20
• Study Start: 2022-01-13
• Primary Completion: 2022-11-15
• Study Completion: 2022-12-15
• Status Verified: 2024-12
• Results First Submitted: 2024-12-23
• Results First Submitted QC: 2024-12-23
• Last Update Submitted: 2024-12-23
• Results First Posted: 2025-01-17
• Last Update Posted: 2025-01-17

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Experimental: PTX-022	PTX-022	PTX-022 QTORIN

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment-Emergent Adverse Events	4 months

Trial Locations

Locations (7)

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

Children's Hospital of Pennsylvania (CHOP); 🇺🇸 Philadelphia, Pennsylvania, United States

Minnesota Clinical Study Center; 🇺🇸 Fridley, Minnesota, United States

Stanford University; 🇺🇸 Palo Alto, California, United States

Cincinnati Children's Hospital; 🇺🇸 Cincinnati, Ohio, United States

Vascular Birthmark Institute; 🇺🇸 New York, New York, United States

Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States