Cell cycle protein-dependent kinase 5 (CDKL5) deficiency (CDD)
Conditions
Interventions
Ganaxolone Oral Suspension:None
Sponsors
children's hospital of fudan university
Eligibility
Sex/Gender
All
Age
2 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. the subject or his/her legal guardian (e.g., parent or legal custodian) has the ability to understand the purpose and risks of the study, to provide signed and dated informed consent, and to authorize permission for the use of protected health information in accordance with state and local regulations related to subject privacy. 2. male or female patients 2 years of age and older with CDD confirmed by molecular diagnosis, with seizures, and with the subject's (or legal guardian's) consent, the investigator decides to prescribe ganaxolone oral suspension treatment for the subject.
Exclusion criteria
Exclusion criteria: 1. the investigator judges that it is not possible to follow the study requirements for receiving treatment, recording relevant information and regular follow-up. 2. current or previous treatment with ganaxolone by commercial routes or in clinical studies. 3. currently participating in other interventional clinical studies. 4. Women who are pregnant or breastfeeding. 5. Known hypersensitivity to ganaxolone and its excipients, or steroidal ingredients such as progesterone. 6. Any other reason not listed here and for which the investigator or sponsor believes the subject is not suitable for enrollment in the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Security Analysis;Validity analysis; Web (AE) Incidence of adverse events (AEs) during the treatment period ? 2025 Microsoft Cookie; | — |
Secondary
| Measure | Time frame |
|---|---|
| Baseline data analysis;; | — |
Countries
China
Contacts
Public ContactYi Wang
children's hospital of fudan university
Outcome results
None listed