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

December 23, 2024 updated by: Palvella Therapeutics, Inc.

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Arkansas Children's Hospital
    • California
      • Palo Alto, California, United States, 94305
        • Stanford University
    • Minnesota
      • Fridley, Minnesota, United States, 55432
        • Minnesota Clinical Study Center
    • New York
      • New York, New York, United States, 10065
        • Vascular Birthmark Institute
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Children's Hospital of Pennsylvania (CHOP)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

13 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental: PTX-022
PTX-022 QTORIN
Drug: PTX-022
Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of Treatment-Emergent Adverse Events
Time Frame: 4 months
4 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change in clinician global assessment from Baseline to Month 4
Time Frame: 4 months
4 months
Change in patient global assessment from Baseline to Month 4
Time Frame: 4 months
4 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Palvella Therapeutics, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

January 13, 2022

Primary Completion (Actual)

November 15, 2022

Study Completion (Actual)

December 15, 2022

Study Registration Dates

First Submitted

September 8, 2021

First Submitted That Met QC Criteria

September 17, 2021

First Posted (Actual)

September 20, 2021

Study Record Updates

Last Update Posted (Actual)

March 25, 2025

Last Update Submitted That Met QC Criteria

December 23, 2024

Last Verified

December 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PALV-06

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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