NCT02160899

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS-APO(a)Rx in Participants With High Lipoprotein(a)

A Randomized, Double Blind, Placebo-Controlled, Dose Titration, Phase 2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 494372 Administered Subcutaneously to Patients With High Lipoprotein(a)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 64 (actual)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

The purpose is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ISIS-APO(a)Rx given to participants with high lipoprotein(a) for 12 weeks.

Detailed description

Lipoprotein(a) \[Lp(a)\] is a genetic variant of low-density lipoprotein (LDL) in which the apolipoprotein B (apoB) -100 component of LDL is linked by a disulfide bond to apolipoprotein(a) \[apo(a)\], the distinct protein component of Lp(a) that is mainly responsible for its signature structural and functional properties. Lp(a) is now recognized as an important genetic risk factor for coronary artery disease, stroke and aortic stenosis. The purpose of this study is to determine if ISIS-APO(a)Rx can reduce the production of apolipoprotein(a), or apo(a). This study will enroll 50 participants with Lipoprotein(a) ≥50 and \<175 mg/dL and 10 participants with Lipoprotein(a) ≥175 mg/dL.

Conditions

• Elevated Lipoprotein(a)

Interventions

Type	Name	Description
DRUG	ISIS-APO(a)Rx	ISIS-APO(a)Rx subcutaneously: 100 mg on Days 1, 8, 15, and 22; 200 mg on Days 29, 36, 43, and 50 unless down-titrated; and 300 mg on Days 57, 64, 71, and 78 unless down-titrated.
DRUG	Placebo	Normal saline as Placebo, subcutaneously on Days 1, 8, 15, 22, 29, 36, 43, 50, 57, 64, 71, and 78.

Timeline

• Start date: 2014-06-01
• Primary completion: 2015-11-01
• Completion: 2015-11-01
• First posted: 2014-06-11
• Last updated: 2019-12-20
• Results posted: 2019-12-20

Locations

13 sites across 5 countries: Canada, Denmark, Germany, Netherlands, United Kingdom