Angelman Syndrome (AS)
Conditions
Brief summary
1. Frequency and severity of adverse events, serious adverse events, treatment discontinuations due to adverse events (MAD, LTE, OOE), 2. Frequency of abnormal laboratory findings (blood and cerebrospinal fluid [CSF]) (MAD, LTE, OOE), 3. Frequency of abnormal vital signs and ECG values (MAD, LTE), 4. Mean changes from baseline in vital signs (temperature, systolic and diastolic blood pressure, heartrate, respiratory rate) over time (MAD, LTE)
Detailed description
1. Time to maximum concentration (Tmax) (MAD, LTE, OOE), 2. Maximum plasma concentration observed (Cmax) (MAD, LTE, OOE), 3. AUC from Time 0 to time of last sampling point or last quantifiable sample, whichever comes first (AUClast), AUC from Time 0 to infinity (AUCinf) (MAD, LTE, OOE)
Interventions
DRUGRugonersen
Sponsors
F. Hoffmann-La Roche AG
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Frequency and severity of adverse events, serious adverse events, treatment discontinuations due to adverse events (MAD, LTE, OOE), 2. Frequency of abnormal laboratory findings (blood and cerebrospinal fluid [CSF]) (MAD, LTE, OOE), 3. Frequency of abnormal vital signs and ECG values (MAD, LTE), 4. Mean changes from baseline in vital signs (temperature, systolic and diastolic blood pressure, heartrate, respiratory rate) over time (MAD, LTE) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Time to maximum concentration (Tmax) (MAD, LTE, OOE), 2. Maximum plasma concentration observed (Cmax) (MAD, LTE, OOE), 3. AUC from Time 0 to time of last sampling point or last quantifiable sample, whichever comes first (AUClast), AUC from Time 0 to infinity (AUCinf) (MAD, LTE, OOE) | — |
Countries
Italy, Netherlands, Spain
Outcome results
None listed