Conditions: Microcystic Lymphatic Malformation
Allocation: NA | Intervention model: SINGLE_GROUP
Enrollment: 12
Healthy volunteers: no

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:

Arms:
arm 1: PTX-022 QTORIN

Interventions: intervention 1: PTX-022
Intervention descriptions: intervention 1: Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations

Protocol document:
Prot_SAP_000.pdf: 
PTX-022 
Palvella Therapeutics, Inc. 
Protocol PALV-06
27June2022 
Amendment #4 
Confidential 
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  
Protocol Number: PALV-06 
Compound Number 
PTX-022 
Sponsor Name: 
Palvella Therapeutics, Inc. 
Legal Registered Address: 
125 Strafford Ave, Suite 360 
Wayne, PA 19087 
Regulatory Agency Identifying 
Number(s): 
Approval Date 
27June2022