Phase 2
Completed N=9
A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Homozygous Familial Hypercholesterolemia
Source: ClinicalTrials.gov NCT02963311 ↗
Enrolled (actual)
9
Serious AEs
25.0%
Results posted
May 2020
Primary outcomePrimary: Percentage Change From Day 1 to Day 90 in LDL-C — -12.26 percent change
Summary
The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage Change From Day 1 to Day 90 in LDL-C |
-12.26 | — |
| PRIMARY Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C |
-20.96 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in LDL-C |
-56.3; -105.3 | — |
| SECONDARY Percentage Change From Day 1 to Day 60 and to Day 90 in PCSK9 |
-64.9; -59.0 | — |
| SECONDARY Absolute Change From Day 1 to Day 60 and to Day 90 in PCSK9 |
-654.1; -602.3 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol |
-13.9; -19.8 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol |
-77.8; -118.0 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides |
-21.0; -20.13 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides |
-35.5; -26.3 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C |
-12.1; -19.8 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C |
-6.0; -9.5 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C |
-13.9; -19.7 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C |
-71.8; -108.5 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C |
8.5; 55.5 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C |
-15.5; -3.3 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1 |
-8.3; -14.2 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1 |
-11.5; -19.8 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B |
-26.6; -25.0 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B |
-95.3; -86.3 | — |
| SECONDARY Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a |
-3.5; -11.8 | — |
| SECONDARY Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a |
13.5; -23.8 | — |
Eligibility Criteria
Inclusion Criteria
- Males and females, ≥12 years of age with a diagnosis of homozygous familial hypercholesterolemia by genetic confirmation or a clinical diagnosis based on a history of an untreated low-density lipoprotein cholesterol (LDL-C) concentration >500 mg/deciliter (dL) [13 millimoles/liter (mmol/L)] together with either xanthoma before 10 years of age or evidence of heterozygous familial hypercholesterolemia in both parents.
- Stable on a low-fat diet.
- Stable on pre-existing, lipid-lowering therapies (such as statins, cholesterolabsorption inhibitors, bile-acid sequestrants, or combinations thereof) for at least 4 weeks with no planned medication or dose change for the duration of study participation.
- Fasting central lab LDL-C concentration >130 mg/dL (3.4 mmol/L) and triglyceride concentration <400 mg/dL (4.5 mmol/L).
- Body weight of 40 kilograms (kg) or greater at screening.
Exclusion Criteria
- LDL or plasma apheresis within 8 weeks prior to the screening visit, and no plan to receive it during the study because of the attendant difficulty in maintaining stable concentrations of LDL-C while receiving apheresis.
- Use of mipomersen or lomitapide therapy within 5 months of screening.
- Previous treatment with monoclonal antibodies directed towards PCSK9 within 8 weeks of screening.
The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
Data sourced from ClinicalTrials.gov (NCT02963311). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.