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Clinical Trials/NCT02963311
NCT02963311CompletedPhase 2

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

The Medicines Company3 sites in 3 countries9 target enrollmentStarted: December 13, 2016Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Completed
Enrollment
9
Locations
3
Primary Endpoint
Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

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

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
12 Years to — (Child, Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

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.

Arms & Interventions

ALN-PCSSC

Experimental

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Intervention: ALN-PCSSC (Drug)

ALN-PCSSC

Experimental

300 milligrams (mg) administered subcutaneous (SC) on Day 1. Participants with a mean serum proprotein convertase subtilisin/kexin type 9 (PCSK9) levels not suppressed by >70% at Day 60 or Day 90, as compared to baseline, will receive a second dose at Day 90 or Day 104, respectively, based on PCSK9 levels from the previous visit. Participants also received standard of care as background therapy.

Intervention: Standard of Care (Drug)

Outcomes

Primary Outcomes

Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C

Time Frame: Day 1, Day 180 (or Final Visit)

Percentage Change From Day 1 to Day 90 in LDL-C

Time Frame: Day 1, Day 90

Due to the small number of subjects, and the fact that the data are not normally distributed, the data are presented as descriptive statistics with no inferential and limited summary statistics presented.

Secondary Outcomes

  • Absolute Change From Day 1 to Day 60 and to Day 90 in PCSK9(Day 1, Day 60, Day 90)
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 60 and to Day 90 in PCSK9(Day 1, Day 60, Day 90)
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein B(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Lipoprotein-a(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Triglycerides(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Apolipoprotein A1(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in LDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in HDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in VLDL-C(Day 1, Day 90, Day 180 (or Final Visit))
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Non-HDL-C(Day 1, Day 90, Day 180 (or Final Visit))

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (3)

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