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A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)

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ClinicalTrials.gov Identifier: NCT02314442
Recruitment Status : Completed
First Posted : December 11, 2014
Last Update Posted : December 17, 2015
Sponsor:
Collaborator:
The Medicines Company
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE December 5, 2014
First Posted Date  ICMJE December 11, 2014
Last Update Posted Date December 17, 2015
Study Start Date  ICMJE December 2014
Actual Primary Completion Date May 2015   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 16, 2015)
The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation [ Time Frame: Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose ]
Original Primary Outcome Measures  ICMJE
 (submitted: December 9, 2014)
The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation [ Time Frame: Part 1 (SAD phase): through day 56; Part 2 (MD) phase: through Day 84 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 16, 2015)
  • The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2) [ Time Frame: Part 1 (SAD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 42, 56 days post-dose; Part 2 (MD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 29, 35, 42, 56, 84 days post-dose ]
  • The effect of ALN-PCSSC on serum levels of LDL-C [ Time Frame: Through the sooner of final follow up visit or 180 days post final dose ]
  • The effect of ALN-PCSSC on plasma levels of PCSK9 [ Time Frame: Through the sooner of final follow up visit or 180 days post final dose ]
Original Secondary Outcome Measures  ICMJE
 (submitted: December 9, 2014)
  • The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2) [ Time Frame: Part 1 (SAD) phase: through day 56; Part 2 (MD) phase: through Day 84 ]
  • The effect of ALN-PCSSC on serum levels of LDL-C [ Time Frame: Part 1 (SAD) phase: through day 56; Part 2 (MD) phase: through Day 84 ]
  • The effect of ALN-PCSSC on plasma levels of PCSK9 [ Time Frame: Part 1 (SAD) phase: through day 56; Part 2 (MD) phase: through Day 84 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)
Official Title  ICMJE A Phase 1, Randomized, Single Blind, Placebo Controlled, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN PCSSC in Subjects With Elevated Low Density Lipoprotein Cholesterol
Brief Summary The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-PCSSC in subjects with elevated LDL-C.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Primary Purpose: Treatment
Condition  ICMJE Hypercholesterolemia
Intervention  ICMJE
  • Drug: ALN-PCSSC
    Single or multiple doses of ALN-PCSSC by subcutaneous (sc) injection
  • Drug: Sterile Normal Saline (0.9% NaCl)
    calculated volume to match active comparator
Study Arms  ICMJE
  • Active Comparator: ALN-PCSSC
    Intervention: Drug: ALN-PCSSC
  • Placebo Comparator: Sterile Normal Saline (0.9% NaCl)
    Intervention: Drug: Sterile Normal Saline (0.9% NaCl)
Publications * Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. A Highly Durable RNAi Therapeutic Inhibitor of PCSK9. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13.

*   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: December 16, 2015)
70
Original Estimated Enrollment  ICMJE
 (submitted: December 9, 2014)
76
Actual Study Completion Date  ICMJE November 2015
Actual Primary Completion Date May 2015   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Adequate complete blood counts, liver and renal function
  • Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
  • Male subjects agree to use appropriate contraception
  • Willing to provide written informed consent and willing to comply with study requirements.
  • Non-smokers and non-nicotine users for at least 90 days before screening
  • On stable statin co-medication [for designated multiple dose cohorts only]

Exclusion Criteria:

  • Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
  • Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
  • History of multiple drug allergies or intolerance to subcutaneous injection
  • Received any medication or nutraceutical to alter serum lipids within 30 days before screening (non-statin cohorts only)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02314442
Other Study ID Numbers  ICMJE ALN-PCSSC-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Alnylam Pharmaceuticals
Study Sponsor  ICMJE Alnylam Pharmaceuticals
Collaborators  ICMJE The Medicines Company
Investigators  ICMJE
Study Director: Robert Kauffman, MD Alnylam Pharmaceuticals
PRS Account Alnylam Pharmaceuticals
Verification Date December 2015

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