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

Palvella Therapeutics

Status and phase: Completed; Phase 2

Conditions: Microcystic Lymphatic Malformation

Treatments: Drug: PTX-022

Study type: Interventional

Funder types: Industry

Identifiers

NCT05050149

PALV-06

Details and patient eligibility

About

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.

Enrollment: 12 patients

Sex: All

Ages: 6+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

Experimental: PTX-022

Experimental group

Description:

PTX-022 QTORIN

Treatment:

Drug: PTX-022