NCT02963311

A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)

An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: The Medicines Company · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.

Conditions

• Homozygous Familial Hypercholesterolemia

Interventions

Type	Name	Description
DRUG	ALN-PCSSC	ALN-PCSSC is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.
DRUG	Standard of Care	Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.

Timeline

• Start date: 2016-12-13
• Primary completion: 2018-10-08
• Completion: 2018-10-08
• First posted: 2016-11-15
• Last updated: 2020-05-18
• Results posted: 2020-05-18

Locations

3 sites across 3 countries: United States, Netherlands, South Africa

Regulatory

• FDA-regulated drug study