A Phase 2 Open-Label Study to Evaluate the Safety and Efficacy of DCR-PHXC in Patients With Primary Hyperoxaluria Type 1 or 2 and Severe Renal Impairment, With or Without Dialysis

Phase 2

• Conditions: Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney DiseasesUrologic DiseasesGenetic Disease; N/A; T51.2

• Registration Number: LBCTR2022125202
• Lead Sponsor: Dicerna Pharmaceuticals

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-02-29
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Pending
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Key inclusion criteria include:
•Genetically confirmed PH1 or PH2
•Estimated glomerular filtration rate (eGFR) < 30 mL/min normalized to 1.73 m2 body surface area (BSA)
•Mean of 2 Pox values > 20 µmol/L during Screening
•For participants receiving hemodialysis or peritoneal dialysis, total duration of hemodialysis or peritoneal dialysis must be less than or equal to 18 months and stable dialysis regimen for at least 2 weeks prior to Screening
•Willing to adhere to a low oxalate diet and avoid high doses of vitamin C

Exclusion Criteria

Key exclusion criteria include:
•Hepatic transplantation, prior to or planned in the 6 months from Day 1. Renal transplantation planned in the 6 months from Day 1. Prior renal transplantation is allowed.
•Documented evidence of clinical manifestations of systemic oxalosis

Study & Design

• Study Type: Interventional
• Study Design: Not specified