The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.
This is an open-label, repeat-dose, Phase 2 study of nedosiran in participants 11 years of age or younger who have PH1, PH2 or PH 3 and relatively intact renal function. Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. The total duration of this study is approximately 15 months from first participant, first visit, until last participant, last visit.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
27
Monthly subcutaneous dosing throughout study period
Clinical Research Site
Rochester, Minnesota, United States
Clinical Research Site
Hamilton, Ontario, Canada
Clinical Trial Site
Bonn, Germany
Efficacy: Percent change in urinary oxalate to creatinine ratio
Evaluate the effect of nedosiran on percent change in urinary oxalate-to-creatinine ratio
Time frame: 180 days
Efficacy: Absolute change in urinary oxalate to creatinine ratio
Evaluate the effect of nedosiran on absolute change in urinary oxalate-to-creatinine ratio
Time frame: 180 days
Safety: Incidence of Events
Characterize the safety of nedosiran in children 11 years of age and younger based on treatment emergent adverse events, and serious adverse events.
Time frame: 180 days
Safety: Changes from baseline in ECG: Rhythm
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in ECG.
Time frame: 180 days
Safety: Changes from baseline in ECG: Ventricular Rate
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in ECG.
Time frame: 180 days
Safety: Changes from baseline in ECG: PR interval
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in ECG.
Time frame: 180 days
Safety: Changes from baseline in ECG: QRS duration
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in ECG.
Time frame: 180 days
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Clinical Research Site
Heidelberg, Germany
Clinical Trial Site
Roma, Italy
Clinical Research Site
Fukuoka, Japan
Clinical Research Site
Nagoya, Japan
Clinical Research Site
Beirut, Lebanon
Clinical Research Site
Bialystok, Poland
Clinical Research Site
Barcelona, Spain
...and 3 more locations
Safety: Changes from baseline in ECG: QT interval
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in ECG.
Time frame: 180 days
Safety: Changes from baseline: Physical Exam
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in physical exam and physician review of systems based on CTCAE v5.0
Time frame: 180 days
Safety: Changes from baseline in Vitals: temperature
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in vital signs.
Time frame: 180 days
Safety: Changes from baseline in Vitals: pulse rate
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in vital signs.
Time frame: 180 days
Safety: Changes from baseline in Vitals: respiratory rate
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in vital signs.
Time frame: 180 days
Safety: Changes from baseline in Vitals: blood pressure
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in vital signs.
Time frame: 180 days
Safety: Changes from baseline: Labs - Hematology
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Clinical Chemistry
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Coagulation
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Antibody
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Plasma Oxalate
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Urinalysis
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
Safety: Changes from baseline: Labs - Urine Oxalate
Characterize the safety of nedosiran in children 11 years of age and younger changes from baseline in clinical laboratory tests.
Time frame: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing secondary parameters of the area under the curve.
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including secondary parameters of area under the curve (AUC)
Time frame: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum observed concentration (Cmax).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including maximum observed concentration (Cmax)
Time frame: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing minimum concentration (Cmin).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including minimum observed concentration (Cmin)
Time frame: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing maximum concentration (Tmax).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including time to maximum concentration (Tmax)
Time frame: 180 days
To characterize the PK of DCR PHXC in patients with PH by observing terminal elimination half-life (t1/2).
Population and individual pharmacokinetic (PK) parameters for DCR PHXC, including terminal elimination half-life (t1/2)
Time frame: 180 days
Efficacy: eGFR changes
Evaluate the effect of nedosiran on eGFR from baseline
Time frame: 180 days