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

Sponsor

Palvella Therapeutics, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05050149

Collaborator

(none)

Enrollment

Locations

Arm

10.4

Anticipated Duration (Months)

2.5

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Microcystic Lymphatic Malformation	Drug: PTX-022	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

15 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Intervention Model Description:

This is an open-label studyThis 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 :

Sep 15, 2021

Anticipated Primary Completion Date :

Apr 30, 2022

Anticipated Study Completion Date :

Jul 30, 2022

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Incidence of Treatment-Emergent Adverse Events [4 months]

Secondary Outcome Measures

Change in clinician global assessment from Baseline to Month 4 [4 months]
Change in patient global assessment from Baseline to Month 4 [4 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

13 Years and Older

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Arkansas Children's Hospital	Little Rock	Arkansas	United States	72202
2	Stanford University	Palo Alto	California	United States	94305
3	Minnesota Clinical Study Center	Fridley	Minnesota	United States	55432
4	Cincinnati Children's Hospital	Cincinnati	Ohio	United States	45229
5	Oregon Health and Science University	Portland	Oregon	United States	97239
6	Children's Hospital of Pennsylvania (CHOP)	Philadelphia	Pennsylvania	United States	19104

Sponsors and Collaborators

Palvella Therapeutics, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Palvella Therapeutics, Inc.

ClinicalTrials.gov Identifier:

NCT05050149

Other Study ID Numbers:

PALV-06

First Posted:

Sep 20, 2021

Last Update Posted:

Mar 2, 2022

Last Verified:

Feb 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Lymphangioma

Lymphatic Abnormalities

Congenital Abnormalities

Study Results

No Results Posted as of Mar 2, 2022