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Trial record 4 of 73 for:    lymphatic malformation

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05050149
Recruitment Status : Recruiting
First Posted : September 20, 2021
Last Update Posted : August 29, 2022
Information provided by (Responsible Party):
Palvella Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
Microcystic Lymphatic Malformation Drug: PTX-022 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: This is an open-label study
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : September 15, 2021
Estimated Primary Completion Date : November 15, 2022
Estimated Study Completion Date : December 15, 2022

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

Primary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [ Time Frame: 4 months ]

Secondary Outcome Measures :
  1. Change in clinician global assessment from Baseline to Month 4 [ Time Frame: 4 months ]
  2. Change in patient global assessment from Baseline to Month 4 [ Time Frame: 4 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05050149

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Contact: Emily Cook 484-253-1463 emily.cook@palvellatx.com

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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
Contact: Jeff Flowers       FlowersJB@archildrens.org   
United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94305
Contact: Elidia Tafoya       etafoya@stanford.edu   
United States, Minnesota
Minnesota Clinical Study Center Recruiting
Fridley, Minnesota, United States, 55432
United States, New York
Vascular Birthmark Institute Recruiting
New York, New York, United States, 10065
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States, 97239
Contact: Chloe Vignola    503-418-9045    vignola@ohsu.edu   
United States, Pennsylvania
Children's Hospital of Pennsylvania (CHOP) Recruiting
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Palvella Therapeutics, Inc.
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Palvella Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05050149    
Other Study ID Numbers: PALV-06
First Posted: September 20, 2021    Key Record Dates
Last Update Posted: August 29, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Lymphatic Abnormalities
Congenital Abnormalities
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Lymphatic Diseases