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Trial record 1 of 1 for:    ORION-2
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A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH) (ORION-2)

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ClinicalTrials.gov Identifier: NCT02963311
Recruitment Status : Completed
First Posted : November 15, 2016
Last Update Posted : December 21, 2018
Sponsor:
Information provided by (Responsible Party):
The Medicines Company

Tracking Information
First Submitted Date  ICMJE November 10, 2016
First Posted Date  ICMJE November 15, 2016
Last Update Posted Date December 21, 2018
Actual Study Start Date  ICMJE December 13, 2016
Actual Primary Completion Date October 8, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 10, 2016)
  • Percentage Change From Day 1 to Day 90 in LDL-C [ Time Frame: Day 1, Day 90 ]
  • Percentage Change From Day 1 to Day 180 (or Final Visit) in LDL-C [ Time Frame: Day 1, Day 180 (or Final Visit) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: November 10, 2016)
  • Absolute Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in LDL-C [ Time Frame: Day 1, Day 90, Day 180 (or Final Visit) ]
  • Percentage Change From Day 1 to Day 60 and to Day 90 in PCSK9 [ Time Frame: Day 1, Day 60, Day 90 ]
  • Absolute Change From Day 1 to Day 60 and to Day 90 in PCSK9 [ Time Frame: Day 1, Day 60, Day 90 ]
  • Percentage Change From Day 1 to Day 90 and to Day 180 (or Final Visit) in Total Cholesterol, Triglycerides, HDLC, non-HDL-C, VLDL-C, Apo-A1 Apo-B and Lp(a) [ Time Frame: 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 Total Cholesterol, Triglycerides, HDLC, non-HDL-C, VLDL-C, Apo-A1 Apo-B and Lp(a) [ Time Frame: Day 1, Day 90, Day 180 (or Final Visit) ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of ALN-PCSSC in Participants With Homozygous Familial Hypercholesterolemia (HoFH)
Official Title  ICMJE An Open-Label, Single-Arm, Multicenter Pilot Study to Evaluate Safety, Tolerability, and Efficacy of ALN-PCSSC in Subjects With Homozygous Familial Hypercholesterolemia (HoFH)
Brief Summary The purpose of this study is to assess the safety, tolerability, and efficacy of ALN-PCSSC in participants with homozygous familial hypercholesterolemia.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Homozygous Familial Hypercholesterolemia
Intervention  ICMJE
  • Drug: ALN-PCSSC
    ALN-PCSSC is a small interfering ribonucleic acid (siRNA) that inhibits PCSK9 synthesis and is given as SC injections.
    Other Name: PCSK9 synthesis inhibitor
  • Drug: Standard of Care
    Included maximally-tolerated statin therapy and/or other low density lipoprotein-cholesterol (LDL-C)-lowering therapies.
Study Arms  ICMJE Experimental: ALN-PCSSC
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.
Interventions:
  • Drug: ALN-PCSSC
  • Drug: Standard of Care
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 23, 2018)
4
Original Estimated Enrollment  ICMJE
 (submitted: November 10, 2016)
10
Actual Study Completion Date  ICMJE October 8, 2018
Actual Primary Completion Date October 8, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Netherlands,   South Africa,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02963311
Other Study ID Numbers  ICMJE MDCO-PCS-16-02
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party The Medicines Company
Study Sponsor  ICMJE The Medicines Company
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Kees Hovingh, MD, PhD Department of Vascular Medicine, Academic Medical Center
PRS Account The Medicines Company
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP