Long Term Extension Study in Patients With Primary Hyperoxaluria

• Status: Enrolling by invitation
• Trial end date: 2023-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dicerna Pharmaceuticals, Inc.

Criteria

Key Inclusion Criteria:

•Participant successfully completed a Dicerna Pharmaceuticals, Inc. study of DCR PHXC.

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.

• 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.

• Estimated GFR at screening ≥ 30 mL/min normalized to 1.73 m2 body surface area (BSA), calculated using Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula in participants aged ≥ 18 years (Levey & Stevens, 2010), or the multivariate equation by Schwartz in participants aged 6 to 17 years (Schwartz et al., 2012). In Japan, the formula by Uemura et al. will be used for participants aged 6 to 17 years, and the equation by Matsuo et al. will be used in participants aged ≥ 18 years (Uemura et al., 2014; Matsuo et al., 2009).

Key Exclusion Criteria:

• Renal or hepatic transplantation (prior or planned within the study period)

• Plasma oxalate > 30 µmol/L

• Currently dialysis

• Documented evidence of clinical manifestations of systemic oxalosis