GNAO1 encephalopathy
Conditions
Interventions
Sponsors
Eligibility
Inclusion criteria
Inclusion criteria: 1. Gender is not limited, age > 1 year old, =6 months, in principle, do not change the pre-enrollment treatment plan, and can be followed up for a long time; 5. The child's caregiver (parent/legal guardian/other entrusted caregiver) is willing and able to comply with all study requirements, including continuously filling out a seizure diary, etc.
Exclusion criteria
Exclusion criteria: 1. Patients with severe physical diseases and severe liver and kidney insufficiency; 2. Patients with clinically significant psychiatric illness, psychological or behavioral problems that may affect the study; 3. Those who cannot follow up regularly; 4. Special populations (allergic to the drug, participation in other clinical trials in the past 30 days).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The form and frequency of epilepsy and movement disorders;Adverse effects; | — |
Secondary
| Measure | Time frame |
|---|---|
| GMFM scale score;AIMS scale score; | — |
Countries
China
Contacts
Shenzhen Children’s Hospital