Clinical Studies for Microcystic Lymphatic Malformations
Our multicenter phase 2 study in the United States evaluating the safety and effectiveness of QTORIN™ rapamycin 3.9% in adolescents and adults age 13 and older with microcystic lymphatic malformations is now complete.
Please use the link below to learn about upcoming studies.
Palvella has recently completed a multi-center phase 2 open label clinical study evaluating the safety and effectiveness QTORIN™ rapamycin 3.9% for the treatment of microcystic lymphatic malformations. Having worked closely with patient advocacy groups to identify the most burdensome symptoms of microcystic lymphatic malformations, the clinical study was designed to determine if QTORIN™ rapamycin 3.9% rapamycin is safe and effective in treating key symptoms of microcystic lymphatic malformations including leaking and bleeding.
Microcystic Lymphatic Malformations (Micro LM) is a rare genetic disease of the lymphatic system driven by a genetic defect in the PI3K/AKT/mTOR pathway. Micro LMs often have a cutaneous component and clinically present with lymphorrhea, bleeding, pain, and functional deficits. Infections of malformations can occur and may lead to cellulitis of surrounding tissues or severe, life-threatening infections. While most individuals are diagnosed early in life, the disease can become more pronounced as an individual ages and goes through puberty.
There are no FDA approved treatment for Microcystic Lymphatic Malformations.
About QTORIN rapamycin 3.9%
QTORIN™ rapamycin 3.9% is a novel targeted topical formulation of rapamycin , an mTOR inhibitor, being studied for Microcystic Lymphatic Malformations a serious and lifelong genetic disease with no FDA approved therapies.
QTORIN™ rapamycin 3.9% has been awarded both FDA Orphan Drug Designation and Fast Track Designation for its Microcystic Lymphatic Malformations research program.
To learn more about participating in upcoming studies, please complete the form below.
Our studies typically pay for costs related to travel for you and a caregiver. During these studies, we are mindful of the impact COVID-19 on the health of our participants and clinicians.
Deciding whether to participate in a clinical trial is a voluntary and personal decision. We encourage you to talk with your family about whether enrolling in any clinical trial is the right decision for you.
Requirements May Include:
Clinically confirmed diagnosis of Microcystic Lymphatic Malformation
Age 13 years or older
If less <18 years of age, the participant and legal guardian must provide written informed consent/assent prior to any study procedures.
The microcystic LM must have a defined area of >~25 cm2, with no more than three total areas to treat. The total area cannot be > ~200 cm2
Able and willing to comply with all protocol-requiring activities