Overview

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