At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 9 enrolled
Drug / intervention
ALN-PCSSC +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
In Brief
A Phase 2 clinical trial evaluating ALN-PCSSC and Standard of Care for Homozygous Familial Hypercholesterolemia. Completed, enrolled 9 participants across 3 sites in 3 countries.
Detailed Summary
The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsHomozygous Familial Hypercholesterolemia
CountriesNetherlands, South Africa, United States
Collaborators--
Timeline
Phase 2CompletedFinished
2017201820192020202120222023202420252026
First PostedNov 2016
Enrollment StartDec 2016
Primary CompletionOct 2018
TodayJul 2026
First PostedNov 15, 2016
Enrollment StartDec 13, 2016
Primary CompletionOct 8, 2018
TodayJul 2, 2026
Enrollment to primary: 1.8 yearsPosted 9.6 years ago
Interventions
ALN-PCSSCdrug
ALN-PCSSC is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.
Standard of Caredrug
Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.