A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904

NCT04731623 | Completed Phase 1 FDA Drug

• 1 other identifier: ION904-CS1
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the safety and tolerability of ION904.

Conditions

Healthy Volunteers

Keywords

Healthy Participants; Hypertension

Outcome Measures

Primary Outcomes (4)

• Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)

  Up to Day 85

• Severity and dose relationship of TEAE

  Up to Day 85

• Severity and dose relationship of clinically significant changes in laboratory values

  Up to Day 85

• Severity and dose relationship of clinically significant changes in ECG

  Up to Day 85

Secondary Outcomes (6)

• Changes in the Level of ION904 Target Biomarker

  Up to Day 85

• Cmax: Maximum Observed Plasma Concentration of ION904

  Up to Day 85

• Tmax: Time to Reach the Maximum Plasma Concentration of ION904

  Up to Day 85

• t½λz: Plasma Elimination Half-life of ION904

  Up to Day 85

• AUC0-t: Area Under the Plasma Concentration-Time Curve from Time Zero to Time t for ION904

  Up to Day 85

Study Arms (2)

ION904

EXPERIMENTAL

Single ascending dose of ION904 will be administered by SC injection on Day 1.

Drug: ION904

Placebo

PLACEBO COMPARATOR

Placebo (0.9% sterile saline) will be administered by SC injection on Day 1.

Drug: Placebo

Interventions

ION904 DRUG

ION904 will be administered by SC injection.

ION904

Placebo DRUG

Placebo (0.9% sterile saline) will be administered by SC injection.

Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Must be healthy males, or females of non-childbearing potential, aged 18 to 65 at the time of informed consent
• Females must be either surgically sterile or post-menopausal; males must be abstinent, surgically sterile, or if engaged in sexual relations with a woman of childbearing potential, a highly effective contraceptive method must be used from the time of signing the informed consent form until at least 13 weeks after the last dose of Study Drug or the healthy participant must be abstinent through this time period
• Willing to refrain from strenuous exercise/activity (for example heavy lifting, weight training, intense aerobics classes etc.) for at least 24 hours prior to study visits
• Body mass index (BMI) requirements between 18 and 35 kg/m\^2

You may not qualify if:

• Clinically significant abnormalities in medical history or physical examination
• Uncontrolled arterial hypertension
• Active infection with human immunodeficiency virus (HIV), hepatitis C or hepatitis B or prior treatment for hepatitis C
• Treatment with another investigational drug, biological agent, or device within 1 month of Screening
• History of bleeding diathesis or coagulopathy
• Regular use of alcohol within 6 months prior to screening, or use of soft drugs within 3 months prior to Screening, or hard drugs within 1 year prior to Screening, or positive urine drug screen at Screening
• History of any severe (e.g., anaphylaxis) drug allergies
• History of hypersensitivity to other antisense oligonucleotides (ASOs)
• Concomitant medication restrictions: over-the-counter or prescription medications within 14 days, prior to dosing on Day 1. Accepted exceptions are:
• Acetaminophen (paracetamol) up to 2 grams per day (g/d) or ibuprofen up to 2.4 g/day taken sporadically for analgesia
• Thyroid hormone replacement therapy provided participant is stable on replacement therapy (and participant is euthyroid)
• Other possible exceptions must be reviewed and acknowledged by the Investigator in consultation with Sponsor's Medical Monitor
• Blood donation of 50 to 499 mL within 30 days of Screening or of \> 499 mL within 60 days of Screening

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (1)

BioPharma Services, Inc.

Toronto, Ontario, M9L 3A2, Canada

Location

MeSH Terms

Conditions

Hypertension

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2021
• First Posted: February 1, 2021
• Study Start: February 26, 2021
• Primary Completion: August 12, 2021
• Study Completion: September 11, 2021
• Last Updated: August 9, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)