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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-PKK-LRx Administered Subcutaneously to Healthy Volunteers

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03263507
Recruitment Status : Completed
First Posted : August 28, 2017
Last Update Posted : February 12, 2020
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE August 17, 2017
First Posted Date  ICMJE August 28, 2017
Last Update Posted Date February 12, 2020
Actual Study Start Date  ICMJE August 28, 2017
Actual Primary Completion Date October 9, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 23, 2017)
  • Incidence and severity of adverse events that are related to treatment with IONIS-PKK-LRx [ Time Frame: Up to 176 days ]
    The safety and tolerability of multiple doses of IONIS-PKK-LRx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS-PKK-LRx.
  • Peak plasma Concentration (Cmax) of IONIS-PKK-LRx [ Time Frame: Up to 176 days ]
    Maximum IONIS-PKK-LRx plasma concentration, Cmax (ug/mL) will be assessed following SC administration
  • Time to peak plasma Concentration (Tmax) of IONIS-PKK-LRx [ Time Frame: Up to 176 days ]
    Time to peak IONIS-PKK-LRx plasma concentration, Tmax (hours) will be assessed following SC administration
  • Effects of IONIS-PKK-LRx on plasma PKK concentration [ Time Frame: Up to 176 days ]
    Effects of IONIS-PKK-LRx on plasma PKK concentration after multiple doses of IONIS-PKK-Lrx compared to baseline
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-PKK-LRx Administered Subcutaneously to Healthy Volunteers
Official Title  ICMJE A Double-Blind, Placebo-Controlled, Dose-Escalation Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 721744, an Antisense Oligonucleotide Inhibitor of PKK, Administered Subcutaneously to Healthy Volunteers
Brief Summary The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-PKK-LRx administered subcutaneously to Healthy Volunteers
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Healthy Volunteers
Intervention  ICMJE
  • Drug: Placebo
    Saline .9%
  • Drug: IONIS-PKK-LRx
    IONIS-PKK-LRx administered subcutaneously
    Other Name: ISIS 721744
Study Arms  ICMJE
  • Experimental: IONIS-PKK-LRx
    Ascending single and multiple doses of IONIS-PKK-LRx administered subcutaneously
    Intervention: Drug: IONIS-PKK-LRx
  • Placebo Comparator: Placebo (sterile saline 0.9%)
    Calculated volume to match active comparator
    Intervention: Drug: Placebo
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: August 23, 2017)
32
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE October 9, 2018
Actual Primary Completion Date October 9, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Must have given written informed consent and be able to comply with all study requirements
  • Healthy males or females aged 18-65 inclusive at the time of Informed Consent
  • Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal
  • Males must be surgically sterile, abstinent or using an acceptable contraceptive method
  • BMI <35 kg/m2

Exclusion Criteria:

  • Clinically-significant abnormalities in medical history including acute coronary syndrome, major surgery within 3 months of Screening, planned surgery that would occur during the study or physical examination or other screening results such as ECGs at Screening
  • Clinically significant hematologic, chemistry, and urine abnormalities
  • Treatment with another Study Drug, biological agent, or device within 4 weeks of Screening
  • History of bleeding diathesis or coagulopathy
  • Smoking >10 cigarettes per day
  • Considered unsuitable for inclusion by the Principal Investigator
  • Current use of concomitant medications (including herbal or OTC medications) unless approved by Sponsor Medical Monitor
  • Known history or positive test for HIV, hepatitis C or chronic hepatitis B
  • Blood donation within 30 days of screening
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 65 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03263507
Other Study ID Numbers  ICMJE ISIS 721744-CS1
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
Product Manufactured in and Exported from the U.S.: Yes
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ionis Pharmaceuticals, Inc.
Study Sponsor  ICMJE Ionis Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Ionis Pharmaceuticals, Inc.
Verification Date February 2020

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