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

Healthy Volunteers Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03263507
• Other study ID #: ISIS 721744-CS1
• Status: Completed
• Phase: Phase 1
• Start date: August 28, 2017
• Est. completion date: October 9, 2018

Study information

• Verified date: August 2022
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 32
• Est. completion date: October 9, 2018
• Est. primary completion date: October 9, 2018
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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

Related Conditions & MeSH terms

• Healthy Volunteers

Intervention

Drug:
• Placebo
Saline .9%
• Donidalorsen
Donidalorsen administered subcutaneously

Locations

Country	Name	City	State
Canada	Ionis Investigative Site	Toronto	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of adverse events that are related to treatment with Donidalorsen	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.	Up to 176 days
Primary	Peak plasma Concentration (Cmax) of Donidalorsen	Maximum Donidalorsen plasma concentration, Cmax (ug/mL) will be assessed following SC administration	Up to 176 days
Primary	Time to peak plasma Concentration (Tmax) of Donidalorsen	Time to peak Donidalorsen plasma concentration, Tmax (hours) will be assessed following SC administration	Up to 176 days
Primary	Effects of Donidalorsen on plasma PKK concentration	Effects of Donidalorsen on plasma PKK concentration after multiple doses of Donidalorsen compared to baseline	Up to 176 days