The aim of this study is to evaluate DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
The DCR-PHXC-204 study is listed under study number NN7022-8398 in Novo Nordisk systems. This is an open-label, repeat-dose, Phase 2 study of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis. Following the up-to-35- day screening period, participants will return to the clinic for monthly dosing visits through Day 180. Participants successfully completing the Day 180 visit will continue on to an extended follow-up period and receive open-label DCR-PHXC for an additional 5 years, or until DCR-PHXC is commercially available, whichever comes first. As participants in this extended treatment period will return to the clinic only every 3 months, participants and/or their caregivers may be trained in the at-home administration of DCR-PHXC or home health nurses may assist with administration of DCR -PHXC. The total duration of the study is approximately 5 years from first participant, first visit, to last participant, last Day 180 visit, with up to an additional 5 years of extended follow-up.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
28
Monthly dosing throughout study period
Clinical Trial Site
San Francisco, California, United States
ACTIVE_NOT_RECRUITINGClinical Trial Site
Boston, Massachusetts, United States
Safety: Incidence of Events
To assess the efficacy of DCR-PHXC in lowering Pox in participants with PH1 and severe renal impairment, with or without hemodialysis or peritoneal dialysis.
Time frame: 180 days
Safety: Incidence of Events
Characterize the safety of DCR-PHXC in participants with PH1 and severe renal impairment, with or without dialysis.
Time frame: 180 days
Change from Baseline in Plasma Oxalate Concentration
To evaluate the effect of DCR-PHXC on Plasma Oxalate concentration from Baseline to day 180
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).
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Clinical Trial Site
Rochester, Minnesota, United States
RECRUITINGClinical Trial Site
New York, New York, United States
ACTIVE_NOT_RECRUITINGClinical Trial Site
Bron, France
WITHDRAWNClinical Trial Site
Paris, France
WITHDRAWNClinical Trial Site
Bonn, Germany
RECRUITINGClinical Trial Site
Heidelberg, Germany
WITHDRAWNClinical Trial Site
Roma, Italy
WITHDRAWNClinical Trial Site
Beirut, Lebanon
RECRUITING...and 8 more locations
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
Change in Frequency of Dialysis
Change from Baseline in the frequency of dialysis over 180 days
Time frame: 180 days
Change in Duration of Dialysis
Change from Baseline in the duration of dialysis over 180 days
Time frame: 180 days