Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency
Conditions
Brief summary
Change from Baseline over time in plasma PPi concentration through Week 52, Overall survival based on time from date of birth to event of all-cause mortality
Detailed description
Change from Baseline in left ventricular ejection fraction via echocardiography through Week 52, Incidence of heart failure, Change from Baseline in vascular calcification in the coronary arteries and aorta via CT scan through Week 52, Change from Baseline in growth Z-score (body length and weight) through Week 52, Growth velocity, Number of days of mechanical ventilation, Measurement of INZ-701 plasma concentration and specific activity
Interventions
DRUGINZ-701
Sponsors
Inozyme Pharma Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change from Baseline over time in plasma PPi concentration through Week 52, Overall survival based on time from date of birth to event of all-cause mortality | — |
Secondary
| Measure | Time frame |
|---|---|
| Change from Baseline in left ventricular ejection fraction via echocardiography through Week 52, Incidence of heart failure, Change from Baseline in vascular calcification in the coronary arteries and aorta via CT scan through Week 52, Change from Baseline in growth Z-score (body length and weight) through Week 52, Growth velocity, Number of days of mechanical ventilation, Measurement of INZ-701 plasma concentration and specific activity | — |
Countries
France, Hungary, Italy, Spain, Sweden
Outcome results
None listed