Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1

• Status: Completed
• Trial end date: 2019-01-23
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects with primary hyperoxaluria type 1 (PH1). In Part A, single ascending dose (SAD) part, healthy adults were dosed with lumasiran or placebo once. In Part B, multiple ascending doses (MAD) part, patients with primary hyperoxaluria type 1 (PH1) were dosed with lumasiran or placebo. All patients that initially received placebo received lumasiran after completing placebo dosing.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Alnylam Pharmaceuticals

Criteria

Inclusion Criteria for Parts A and B:

• Women of child bearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception.

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

Additional Inclusion Criteria for Part B:

• Confirmation of PH1 disease

• Meet 24 hour urine oxalate excretion requirements

• Estimated glomerular filtration rate (GFR) of >45 mL/min/1.73m^2

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

Exclusion Criteria for Parts A and B:

• Clinically significant health concerns (with the exception of PH1 for patients in Part B)

• Clinically significant electrocardiogram (ECG) abnormalities

• Abnormal for aspartate aminotransferase (AST)/alanine aminotransferase (ALT) and any other clinical safety laboratory result considered clinically significant

• Received an investigational agent within 3 months before the first dose of study drug or are in follow-up of another clinical study

• Known history of allergic reaction to an oligonucleotide or N-acetylgalactosamine (GalNAc)

• History of intolerance to subcutaneous injection