A Pharmacokinetic, Pharmacodynamic, Safety and Tolerability Study of CERC-006 in Adults With Complex Lymphatic Malformations

Inclusion Criteria

• •Participants must fulfill the following requirements to be eligible for the study:
• •1. Participant is 18 to 31 years of age (inclusive) at the time of consent.
• •2. Participant has a verified diagnosis of a complex lymphatic malformation. Other moderate to severe vascular anomalies with associated lymphatic involvement will be considered, with approval by the study medical monitor.
• •3. Participant's complex lymphatic malformation is considered, in the opinion of the investigator, to be moderate to severe.
• •4. Participant has adequate liver function defined as:
• •* Total bilirubin (sum of conjugated and unconjugated) ≤1.5 × upper limit of normal (ULN)
• •* Aspartate transaminase/Alanine aminotransferase (AST/ALT) \<5 × ULN
• •* Serum albumin \> 2 g/dL
• •5. Participant has fasting low-density lipoprotein (LDL) of \<160 mg/dL.
• •6. Participant has adequate bone marrow function defined as:
• •* Peripheral absolute neutrophil count (ANC) \> 1000/µL
• •* Hemoglobin \> 8.0 g/dL
• •* Platelet count ≥ 50,000/µL
• •7. Participant has adequate renal function defined as:
• •• Serum creatinine ≤ 1.5 mg/dL or calculated creatinine clearance of \> 50 mL/min according to the Cockcroft-Gault equation
• •8. Participant has agreed to and met the washout period as follows:
• •* At least 14 days prior to initiation of CERC-006 if receiving sirolimus (also known as rapamycin), mitogen-activated protein kinase (MEK) inhibitors, phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA) inhibitors, interferon alfa 2b, vascular endothelial growth factor receptor 3 (VEGFR-3) inhibitors, and/or other systemic agents targeting lymphatic malformation
• •* At least 7 days prior to initiation of CERC-006 if receiving topical agents targeting lymphatic malformation
• •9. Participant has a Karnofsky performance status of ≥50%.