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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-01-13

Study Completion Date

2022-12-15

Brief Summary

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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.

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Detailed Description

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Conditions

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Microcystic Lymphatic Malformation

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

This is an open-label, baseline-controlled study

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Experimental: PTX-022

PTX-022 QTORIN

Group Type EXPERIMENTAL

PTX-022

Intervention Type DRUG

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

Interventions

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PTX-022

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

Intervention Type DRUG

Eligibility Criteria

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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.

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No