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Phase 2 N=9 Treatment

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

Homozygous Familial Hypercholesterolemia

Enrolled (actual)
9
Serious AEs
25.0%
Results posted
May 2020
Primary outcome: Primary: Percentage Change From Day 1 to Day 90 in LDL-C — -12.26 percent change

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
ALN-PCSSC (Drug); Standard of Care (Drug)
Age
Pediatric, Adult, Older Adult · 12+ yrs
Sex
All
Sponsor
The Medicines Company
Primary completion
Oct 2018

Outcome Measures

OutcomeResultp-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

Summary

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

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.

View full record on ClinicalTrials.gov →

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.

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