Long Term Extension Study in Patients With Primary Hyperoxaluria (PHYOX3)

JRCT ID: jRCT2041200091

Registered date:15/01/2021

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Primary hyperoxaluria
• Date of first enrollment: 18/01/2021
• Target sample size: 5
• Countries of recruitment: France,Japan,Germany,Japan,Netherlands,Japan,United Kingdom,Japan,United States,Japan,Australia,Japan,Lebanon,Japan,Spain,Japan,Italy,Japan,Canada,Japan
• Study type: Interventional
• Intervention(s): In adults and in adolescents (aged 12-17 years) weighing >=50 kg, DCR-PHXC will be administered once monthly at a dose of 170 mg. In adults and adolescents weighing < 50 kg, DCR-PHXC will be administered once monthly at a dose of 136 mg.The dose for participants aged 0 to 11 years will be 3.5 mg/kg monthly, not to exceed 170 mg.

Outcome(s)

• Primary Outcome: The annual rate of decline in eGFR in participants with PH1

Key inclusion & exclusion criteria

• Age minimum: >=
• Age maximum: Not applicable
• Gender: Both

Include criteria

- Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR-PHXC. For participants rolling over from a multidose study of DCR-PHXC, enrollment should occur within a window of 25 to 75 days from the last dose of study intervention.
OR Participant is the sibling of a participant who successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR-PHXC. Siblings must be younger than 18 years of age and must have genetically confirmed PH and two 24-hour Uox values >= 0.7 mmol (adjusted per 1.73 m2 body surface area [BSA]) at screening.
OR
for siblings aged 0 to 5 years old, average spot Uox-to-creatinine ratio at Screening above 2 times the 95th percentile for age based on Matos et al, 1999.
- Estimated GFR at screening >= 30 mL/min normalized to 1.73 m2 body surface area (BSA).
For infants aged less than 12 months, serum creatinine below the 97.5th percentile of a healthy population (Boer et al., 2010).

Exclude criteria

- Renal or hepatic transplantation; prior or planned within the study period
- Plasma oxalate > 30 micromol/L
- Current dialysis
- Documented evidence of clinical manifestations of systemic oxalosis (including pre-existing retinal, heart, or skin calcifications, or history of severe bone pain, pathological fractures, or bone deformations)

Related Information

• Primary Sponsor: Verity Rawson
• Secondary ID(s): NCT04042402