Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04103333 | Angelman Syndrome (AS) Biomarker Study | EARLY_PHASE1 | COMPLETED | 20 | — | — | Dec 18, 2019 | Jan 2, 2022 | Jan 28, 2022 | 5 | United States |
| Arm | Type | Description |
|---|---|---|
| Angelman Syndrome: Group 1 | EXPERIMENTAL | Participants aged 0-6 years old with Angelman Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Angelman Syndrome: Group 2 | EXPERIMENTAL | Participants aged 7-12 years old with Angelman Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Angelman Syndrome: Group 3 | EXPERIMENTAL | Participants aged 13-18 years old with Angelman Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Angelman Syndrome: Group 4 | EXPERIMENTAL | Participants aged 19-50 years old with Angelman Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Dup15q Syndrome: Group 1 | EXPERIMENTAL | Participants aged 0-6 years old with Dup15q Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Dup15q Syndrome: Group 2 | EXPERIMENTAL | Participants aged 7-12 years old with Dup15q Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Dup15q Syndrome: Group 3 | EXPERIMENTAL | Participants aged 13-18 years old with Dup15q Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Dup15q Syndrome: Group 4 | EXPERIMENTAL | Participants aged 19-50 years old with Dup15q Syndrome will undergo lumbar puncture (LP) for CSF and blood collection while under general anesthesia or conscious sedation during or after a prescheduled procedure unrelated to the study. |
| Name | Type | Description |
|---|---|---|
| Lumbar Puncture | PROCEDURE | Administered as specified in the treatment arm. |
| Blood Collection | PROCEDURE | Administered as specified in the treatment arm. |
Key Inclusion Criteria: * Ability of the participant's legally authorized representative (LAR) to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local privacy regula...