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
Why are we doing this research?
The main reason for this research study is to see if a new topical (applied to the skin) formulation of sirolimus, PTX-022, is safe and effective in treating microcystic lymphatic malformation, sometimes called blebs, on the skin. Sirolimus belongs to a group of medicines called mTOR inhibitors, which means that it blocks the action of the mTOR protein. Based on the way it works, PTX-022 is a drug which targets the disease pathway in the skin, and may decrease crusting, leaking, and bleeding, or other symptoms from the lymphatic malformation there.
Who can participate?
You may take part in this study if you are at least 13 years old and have microcystic lymphatic malformation involving the skin and causing some leaking or bleeding.
- Hematology - Hemangioma and Vascular Malformation
What will happen in the study?
You will be treated with PTX-022, spreading it onto a selected area of affected skin once daily, as we assess its safety and effectiveness by looking at pain and symptoms associated with the malformation(s) and your overall health.
We expect that you will be in this research study for up to 20 weeks; however, you will only use the investigational drug for 12 weeks. There will be a screening period of up to 4 weeks between consent and first dose, and a 4-week follow up period after your last dose of investigational drug for safety assessments.
What are the good things that can happen from this research?
PTX-022 may help your pain and symptoms, which may make you feel better; however, taking part in this research study may or may not make your health better. We hope the information learned from this research study will help doctors learn more about PTX-022 as a treatment for patients with microcystic lymphatic malformations in the skin in the future.
What are the bad things that can happen from this research?
The investigational drug used in this study may cause skin irritation or could affect how different parts of your body work such as your liver, kidneys, heart, and blood, causing side effects. These will be followed throughout the study.
Will you/your child be paid to be in this research study?
You will receive up to $750 for taking part in this study. In addition, you may be reimbursed for your travel and meals, if needed, while you are in this research study.