A Phase 3 Open-Label Study of Safety, Pharmacokinetics and Activity of Weekly Subcutaneous Pegzilarginase in Subjects <24 months old with Arginase 1 Deficiency

Conditions

Arginase 1 deficiency (ARG1-D)

Brief summary

Change from baseline in plasma arginine after 12 weeks of pegzilarginase treatment

Detailed description

Safety assessments will include: a. adverse events (AEs), including hypersensitivity reactions (HSRs), injection site reactions (ISRs), and hyperammonaemic events; b. laboratory tests; c. vital signs, physical examinations, growth assessments, and electrocardiograms (ECGs), PK parameters evaluation including half-life (T½), time to maximum observed concentration (Tmax), maximum observed concentration (Cmax), area under the plasma drug concentration-time curve from time 0 to time t (AUC0-t), area under the plasma drug concentration-time curve from time 0 extrapolated to infinite time (AUC0-∞), extravascular clearance (CL/F), and apparent volume of distribution at steady state after non-intravenous administration (Vss/F), PD response evaluation including anti-drug antibodies (ADAs), levels of plasma arginine and ornithine, Changes in physical function after 12 weeks of pegzilarginase treatment as measured by Gross Motor Function Measure (GMFM)-66 Parts A through E, as age appropriate and feasible

Related papers

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Interventions

DRUGLoargys 5 mg/ml solution for injection/infusion

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from baseline in plasma arginine after 12 weeks of pegzilarginase treatment	—

Secondary

Measure	Time frame
Safety assessments will include: a. adverse events (AEs), including hypersensitivity reactions (HSRs), injection site reactions (ISRs), and hyperammonaemic events; b. laboratory tests; c. vital signs, physical examinations, growth assessments, and electrocardiograms (ECGs), PK parameters evaluation including half-life (T½), time to maximum observed concentration (Tmax), maximum observed concentration (Cmax), area under the plasma drug concentration-time curve from time 0 to time t (AUC0-t), area under the plasma drug concentration-time curve from time 0 extrapolated to infinite time (AUC0-∞), extravascular clearance (CL/F), and apparent volume of distribution at steady state after non-intravenous administration (Vss/F), PD response evaluation including anti-drug antibodies (ADAs), levels of plasma arginine and ornithine, Changes in physical function after 12 weeks of pegzilarginase treatment as measured by Gross Motor Function Measure (GMFM)-66 Parts A through E, as age appropriate an	—

Measure

Time frame

Safety assessments will include: a. adverse events (AEs), including hypersensitivity reactions (HSRs), injection site reactions (ISRs), and hyperammonaemic events; b. laboratory tests; c. vital signs, physical examinations, growth assessments, and electrocardiograms (ECGs), PK parameters evaluation including half-life (T½), time to maximum observed concentration (Tmax), maximum observed concentration (Cmax), area under the plasma drug concentration-time curve from time 0 to time t (AUC0-t), area under the plasma drug concentration-time curve from time 0 extrapolated to infinite time (AUC0-∞), extravascular clearance (CL/F), and apparent volume of distribution at steady state after non-intravenous administration (Vss/F), PD response evaluation including anti-drug antibodies (ADAs), levels of plasma arginine and ornithine, Changes in physical function after 12 weeks of pegzilarginase treatment as measured by Gross Motor Function Measure (GMFM)-66 Parts A through E, as age appropriate an

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Countries

Austria, Portugal

Outcome results

None listed