A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria

Phase 2

Completed

Conditions: Hyperoxaluria
10027424
Primary Hyperoxaluria
10021605
10038430

Registration Number: NL-OMON49278

Lead Sponsor: Dicerna Pharmaceuticals Inc

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 6

Inclusion Criteria

Key inclusion criteria:
- 24-hour Uox excretion * *0.7 mmol (adjusted per 1.73 m2 body surface area
[BSA] in participants < 18 years of age) in both collections performed in the
screening period. Of the first 24 participants enrolled, at least 12 (50%) must
have at least one 24 hour Uox excretion * 1.6 mmol (adjusted per 1.73 m2 BSA in
participants aged < 18 years).
- Less than 20% variation between the two 24-hour urinary creatinine excretion
values [mmol/24 hr/kg] derived from the two 24-hour urine collections in the
screening period.
- Estimated GFR at screening * 30 mL/min normalized to 1.73 m2 BSA,
calculated using the CKD-EPI formula in participants aged * 18 years or the
2012 multivariate equation by Schwartz in participants aged 6 to 17 years.

Exclusion Criteria

Key exclusion criteria:
- Prior renal or hepatic transplantation; or planned transplantation within
the study period
- Currently receiving dialysis or anticipating requirement for dialysis during
the study period
- Plasma oxalate > 30 *mol/L
- 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).
- Liver function test (LFT) abnormalities: Alanine aminotransferase (ALT)
and/or aspartate aminotransferase (AST) >1.5 times upper limit of normal (ULN)
for age and gender.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary End point:<br /><br>The proportion of participants with a reduction from baseline in 24-hour Uox of<br /><br>at least 70%, based on a AUC and/or reaching normalization or<br /><br>near-normalization of 24-hour Uox on at least 2 consecutive visits, starting<br /><br>from Day 90. Normalization of Uox is defined as < 0.46 mmol/24 hours;<br /><br>near-normalization is defined as * 0.46 to < 0.60 mmol/24 hours (values<br /><br>adjusted per 1.73 m2 BSA in participants aged < 18 years).</p><br>

Secondary Outcome Measures