CTNNB1 syndrome
Conditions
Brief summary
Incidence, severity, and causality of adverse events and serious adverse events., Occurrence of clinically significant abnormalities in blood (blood count, renal function, liver function, coagulation), urine or CSF compared to baseline., Occurrence of clinically significant changes in cardiorespiratory parameters from baseline., Occurrence of new clinically significant cardiovascular abnormalities on 12-lead electrocardiogram (ECG) or echocardiogram., Occurrence of clinically significant electroencephalographic changes from baseline (EEG)., Occurrence of clinically significant changes in head circumference and significant volumetric changes on MRI brain., Presence of antibodies for AAV9 in serum.
Detailed description
To assess the preliminary efficacy of URBAGEN as a single ICV administration in pediatric patients with CTNNB1 syndrome.
Interventions
DRUGURBAGEN
Sponsors
Fundacija CTNNB1 Ustanova Za Raziskave Na Podrocju Genske Terapije
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, severity, and causality of adverse events and serious adverse events., Occurrence of clinically significant abnormalities in blood (blood count, renal function, liver function, coagulation), urine or CSF compared to baseline., Occurrence of clinically significant changes in cardiorespiratory parameters from baseline., Occurrence of new clinically significant cardiovascular abnormalities on 12-lead electrocardiogram (ECG) or echocardiogram., Occurrence of clinically significant electroencephalographic changes from baseline (EEG)., Occurrence of clinically significant changes in head circumference and significant volumetric changes on MRI brain., Presence of antibodies for AAV9 in serum. | — |
Secondary
| Measure | Time frame |
|---|---|
| To assess the preliminary efficacy of URBAGEN as a single ICV administration in pediatric patients with CTNNB1 syndrome. | — |
Countries
Slovenia
Outcome results
None listed