complex vascular malformation
Conditions
Interventions
Treatment group:Oral trametinib
Sponsors
Beijing Children's Hospital, Capital Medical University
Eligibility
Sex/Gender
All
Age
No minimum to 18 Years
Inclusion criteria
Inclusion criteria: (1) Meet ISSVA (2018) diagnostic criteria for complex vascular diseases; (2) Gender is not limited; patients were enrolled if they were between 2 and 18 years of age; (3) The eligible patients had to harbor mutaions in BRAF, KRAS, NRAS, MEK (MAP2K1/MAP2K2) or other gene upstream of MEK pathway by next-generation sequencing (NGS) with cfDNA or tissue samples; (4) Vascular lesions are involving a large area of body , and the depth exceeds the subcutaneous layer; (5) Higher risk of tissue and organ dysfunction;(6) Stable vital signs; (7) Be able to receive treatment; (8) Parents or legal guardians fully understand the content of this trial, voluntarily participate in and sign the informed consent; (9) Pass the physical examination, Patients were required to have adequate heart, lung, liver and renal function, and an absence of diseases of digestive tract, nervous system, mental and metabolic disorders.
Exclusion criteria
Exclusion criteria: (1) Those whose health examination does not meet the inclusion criteria; (2) Hematological system diseases; (3) Autoimmune diseases; (4) Liver dysfunction; (5) Renal dysfunction; (6) Chronic infectious diseases (such as tuberculosis) with severe infection and active period; (7) Individuals who are allergic to the research drug or excipients; (8) Patients who participated in any other drug clinical study three months prior to the start of the trial; (9) Any systemic medication used to treat vascular malformations within 4 weeks prior to baseline visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Skin color;Skin temperature;arterial blood flow velocity; | — |
Secondary
| Measure | Time frame |
|---|---|
| evascularization; | — |
Countries
China
Contacts
Public ContactBin Zhang
Beijing Children's Hospital, Capital Medical University
Outcome results
None listed