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

NCT05050149 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: PALV-06
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 7

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.

Conditions

Microcystic Lymphatic Malformation

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events

  4 months

Study Arms (1)

Experimental: PTX-022

EXPERIMENTAL

PTX-022 QTORIN

Drug: PTX-022

Interventions

PTX-022 DRUG

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

Experimental: PTX-022

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

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.

Sponsors & Collaborators

• Palvella Therapeutics, Inc.: lead

Study Sites (7)

Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States

Location

Stanford University

Palo Alto, California, 94305, United States

Location

Minnesota Clinical Study Center

Fridley, Minnesota, 55432, United States

Location

Vascular Birthmark Institute

New York, New York, 10065, United States

Location

Cincinnati Children's Hospital

Cincinnati, Ohio, 45229, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Pennsylvania (CHOP)

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (1)

• Teng JMC, Hammill A, Martini J, Treat J. Sirolimus in the Treatment of Microcystic Lymphatic Malformations: A Systematic Review. Lymphat Res Biol. 2023 Apr;21(2):101-110. doi: 10.1089/lrb.2021.0103. Epub 2022 Jul 18.

  PMID: 35852876 DERIVED

MeSH Terms

Conditions

Lymphangioma

Condition Hierarchy (Ancestors)

Neoplasm, Lymphatic Tissue; Neoplasms by Histologic Type; Neoplasms

Results Point of Contact

• Title: Christine Kampf, Vice President of Regulatory Affairs
• Organization: Palvella Therapeutics, Inc.

christine.kampf@palvellatx.com

484-844-0865

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is an open-label, baseline-controlled study

Study Record Dates

• First Submitted: September 8, 2021
• First Posted: September 20, 2021
• Study Start: January 13, 2022
• Primary Completion: November 15, 2022
• Study Completion: December 15, 2022
• Last Updated: January 17, 2025
• Results First Posted: January 17, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)