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OpenTrials
Completed

NCT Number: NCT03263507

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

The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen administered subcutaneously to Healthy Volunteers

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Key information

Conditions

Healthy Volunteers

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Ionis Investigative Site

Toronto, Ontario, M9L 3A2, Canada

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Placebo

Drug

Saline .9%

Donidalorsen

Drug

Donidalorsen administered subcutaneously

Other names: ISIS 721744, IONIS-PKK-LRx

Primary outcomes

  1. Incidence and severity of adverse events that are related to treatment with Donidalorsen

    Time frame: Up to 176 days

    The safety and tolerability of multiple doses of Donidalorsen will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with Donidalorsen.

  2. Peak plasma Concentration (Cmax) of Donidalorsen

    Time frame: Up to 176 days

    Maximum Donidalorsen plasma concentration, Cmax (ug/mL) will be assessed following SC administration

  3. Time to peak plasma Concentration (Tmax) of Donidalorsen

    Time frame: Up to 176 days

    Time to peak Donidalorsen plasma concentration, Tmax (hours) will be assessed following SC administration

  4. Effects of Donidalorsen on plasma PKK concentration

    Time frame: Up to 176 days

    Effects of Donidalorsen on plasma PKK concentration after multiple doses of Donidalorsen compared to baseline

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Aug 28, 2017
Registry last updated
Aug 4, 2022

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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