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

Primary Purpose

Microcystic Lymphatic Malformation

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
PTX-022
Sponsored by
Palvella Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Microcystic Lymphatic Malformation

Eligibility Criteria

13 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Arkansas Children's HospitalRecruiting
  • Stanford UniversityRecruiting
  • Minnesota Clinical Study CenterRecruiting
  • Vascular Birthmark InstituteRecruiting
  • Cincinnati Children's HospitalRecruiting
  • Oregon Health and Science UniversityRecruiting
  • Children's Hospital of Pennsylvania (CHOP)Recruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Experimental: PTX-022

Arm Description

PTX-022 QTORIN

Outcomes

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events

Secondary Outcome Measures

Change in clinician global assessment from Baseline to Month 4
Change in patient global assessment from Baseline to Month 4

Full Information

First Posted
September 8, 2021
Last Updated
August 26, 2022
Sponsor
Palvella Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05050149
Brief Title
Study Evaluating the Safety and Efficacy of PTX-022 (QTORIN Sirolimus) in the Treatment of Microcystic Lymphatic Malformations
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Recruiting
Study Start Date
September 15, 2021 (Actual)
Primary Completion Date
November 15, 2022 (Anticipated)
Study Completion Date
December 15, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Palvella Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Microcystic Lymphatic Malformation

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
This is an open-label study
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Experimental: PTX-022
Arm Type
Experimental
Arm Description
PTX-022 QTORIN
Intervention Type
Drug
Intervention Name(s)
PTX-022
Intervention Description
Safety and Efficacy of PTX-022 in the Treatment of Microcystic Lymphatic Malformations
Primary Outcome Measure Information:
Title
Incidence of Treatment-Emergent Adverse Events
Time Frame
4 months
Secondary Outcome Measure Information:
Title
Change in clinician global assessment from Baseline to Month 4
Time Frame
4 months
Title
Change in patient global assessment from Baseline to Month 4
Time Frame
4 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Emily Cook
Phone
484-253-1463
Email
emily.cook@palvellatx.com
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Jeff Flowers
Email
FlowersJB@archildrens.org
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94305
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Elidia Tafoya
Email
etafoya@stanford.edu
Facility Name
Minnesota Clinical Study Center
City
Fridley
State/Province
Minnesota
ZIP/Postal Code
55432
Country
United States
Individual Site Status
Recruiting
Facility Name
Vascular Birthmark Institute
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Individual Site Status
Recruiting
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Chloe Vignola
Phone
503-418-9045
Email
vignola@ohsu.edu
Facility Name
Children's Hospital of Pennsylvania (CHOP)
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
Citations:
PubMed Identifier
35852876
Citation
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.
Results Reference
derived

Learn more about this trial

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

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