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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.

Study Description
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Brief Summary:
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-PKK-LRx administered subcutaneously to Healthy Volunteers

Condition or disease Intervention/treatment Phase
Healthy Volunteers Drug: Placebo Drug: IONIS-PKK-LRx Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 32 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : August 28, 2017
Actual Primary Completion Date : October 9, 2018
Actual Study Completion Date : October 9, 2018
Arms and Interventions
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Arm Intervention/treatment
Experimental: IONIS-PKK-LRx
Ascending single and multiple doses of IONIS-PKK-LRx administered subcutaneously
 Drug: IONIS-PKK-LRx
IONIS-PKK-LRx administered subcutaneously
Other Name: ISIS 721744
Placebo Comparator: Placebo (sterile saline 0.9%)
Calculated volume to match active comparator
 Drug: Placebo
Saline .9%


Outcome Measures
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Primary Outcome Measures :
  1. 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.

  2. 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

  3. 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

  4. 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


Eligibility Criteria
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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03263507


Locations
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Canada, Ontario
Biopharma Services
Toronto, Ontario, Canada, M9L 3A2
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03263507    
Other Study ID Numbers: ISIS 721744-CS1
First Posted: August 28, 2017    Key Record Dates
Last Update Posted: February 12, 2020
Last Verified: February 2020

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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
Keywords provided by Ionis Pharmaceuticals, Inc.:
IONIS PKK-LRx