NCT02706886

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

A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Type 1

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 52 (actual)
• Sponsor: Alnylam Pharmaceuticals · Industry
• Sex: All
• Age: 6 Years – 64 Years
• Healthy volunteers: Accepted

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.

Conditions

• Primary Hyperoxaluria Type 1 (PH1)

Interventions

Type	Name	Description
DRUG	Lumasiran	Lumasiran will be administered SC once in Part A. In Part B lumasiran will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).
DRUG	Placebo	Matching placebo (sterile saline: 0.9% sodium chloride \[NaCl\]) will be administered SC once in Part A. In Part B matching placebo will be administered SC qM (Days 1, 29 and 57) or q3M (Days 1 and 85).

Timeline

• Start date: 2016-03-08
• Primary completion: 2019-01-23
• Completion: 2019-01-23
• First posted: 2016-03-11
• Last updated: 2020-01-30
• Results posted: 2020-01-30

Locations

9 sites across 5 countries: France, Germany, Israel, Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study