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

Microcystic Lymphatic Malformation Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05050149
• Other study ID #: PALV-06
• Status: Recruiting
• Phase: Phase 2
• Start date: September 15, 2021
• Est. completion date: December 15, 2022

Study information

• Verified date: August 2022
• Source: Palvella Therapeutics, Inc.
• Contact: Emily Cook
• Phone: 484-253-1463
• Email: emily.cook[@]palvellatx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: December 15, 2022
• Est. primary completion date: November 15, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years and older

Eligibility

Inclusion Criteria:

• Participants must be 13 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.

Related Conditions & MeSH terms

• Congenital Abnormalities
• Lymphangioma
• Lymphatic Abnormalities
• Microcystic Lymphatic Malformation

Intervention

Drug:
• PTX-022
Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Palvella Therapeutics, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events		4 months
Secondary	Change in clinician global assessment from Baseline to Month 4		4 months
Secondary	Change in patient global assessment from Baseline to Month 4		4 months