A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1

• Status: Active, not recruiting
• Trial end date: 2024-01-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).

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

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria:

• Willing to provide written informed consent or assent and to comply with study requirements

• Confirmation of PH1 disease

• Meet the 24 hour urine oxalate excretion requirements

• If taking Vitamin B6 (pyridoxine), must have been on stable regimen for at least 90 days

Exclusion Criteria:

• Clinically significant health concerns (with the exception of PH1) or clinical evidence of extrarenal systemic oxalosis

• Clinically significant abnormal laboratory results

• Known active or evidence of HIV or hepatitis B or C infection

• An estimated GFR of < 30 mL/min/1.73m^2 at screening

• Received an investigational agent within 30 days or 5 half-lives before the first dose of study drug or are in follow-up of another clinical study

• History of kidney or liver transplant

• Known history of multiple drug allergies or allergic reaction to an oligonucleotide or GalNAc

• History of intolerance to subcutaneous injection

• Women who are pregnant, planning a pregnancy, or breast-feeding or those of child bearing potential and not willing to use contraception

• History of alcohol abuse within the last 12 months, or unable or unwilling to limit alcohol consumption throughout the study