CClinicalTrials.gg
CompletedNCT04731623Updated Aug 9, 2022

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

A Phase 1 interventional study of ION904 and Placebo in Healthy Volunteers, sponsored by Ionis Pharmaceuticals, Inc.. Completed at 1 site in Canada. Open to participants aged 18 Years to 65 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2022-08-09.

Sponsored by Ionis Pharmaceuticals, Inc. · Phase 1, Interventional, and Treatment

From the registry’s dates

  • Primary completion was Aug 2021, 5 years 1 month ago, and no results have been posted to the registry.
Phase
Phase 1
Study type
Interventional
Enrollment
40
Allocation
Randomized
Ages
18 Years to 65 Years
Sex
All
01

Study summary

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

Read the detailed description

This is a Phase 1, randomized, double-blind, placebo-controlled, single-ascending dose escalation study of ION904 in up to 72 participants. Following a Screening Period of up to 28 days, eligible healthy participants will be randomized to treatment and receive a single, subcutaneous (SC) dose of the assigned study drug. Participants will be followed for up to 13 weeks after dosing.

02

Conditions studied

  • Healthy Volunteers

Keywords

  • Healthy Participants
  • Hypertension
03

In context

Lead sponsor

Ionis Pharmaceuticals, Inc. is the lead sponsor of 116 studies on the registry; 10 are open to participants now.

Of its 48 completed or terminated interventional studies of FDA-regulated products, 21 (44%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
Yes

Inclusion criteria

  1. Must be healthy males, or females of non-childbearing potential, aged 18 to 65 at the time of informed consent
  2. 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
  3. 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
  4. Body mass index (BMI) requirements between 18 and 35 kg/m\^2

Exclusion criteria

Exclusion Criteria:

  1. Clinically significant abnormalities in medical history or physical examination
  2. Uncontrolled arterial hypertension
  3. Active infection with human immunodeficiency virus (HIV), hepatitis C or hepatitis B or prior treatment for hepatitis C
  4. Treatment with another investigational drug, biological agent, or device within 1 month of Screening
  5. History of bleeding diathesis or coagulopathy
  6. 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
  7. History of any severe (e.g., anaphylaxis) drug allergies
  8. History of hypersensitivity to other antisense oligonucleotides (ASOs)
  9. 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
  10. Blood donation of 50 to 499 mL within 30 days of Screening or of > 499 mL within 60 days of Screening
05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
40 participants (actual)

Study arms

  • Experimental
    ION904

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

    Drug: ION904

  • Placebo comparator
    Placebo

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

    Drug: Placebo

Interventions

  • DrugION904

    ION904 will be administered by SC injection.

  • DrugPlacebo

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

06

What researchers measure

Primary outcomes

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

    Time frame: Up to Day 85

  2. Severity and dose relationship of TEAE

    Time frame: Up to Day 85

  3. Severity and dose relationship of clinically significant changes in laboratory values

    Time frame: Up to Day 85

  4. Severity and dose relationship of clinically significant changes in ECG

    Time frame: Up to Day 85

Secondary outcomes

  1. Changes in the Level of ION904 Target Biomarker

    Time frame: Up to Day 85

  2. Cmax: Maximum Observed Plasma Concentration of ION904

    Time frame: Up to Day 85

  3. Tmax: Time to Reach the Maximum Plasma Concentration of ION904

    Time frame: Up to Day 85

  4. t½λz: Plasma Elimination Half-life of ION904

    Time frame: Up to Day 85

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

    Time frame: Up to Day 85

  6. Urine 0-24 Hour (hr) Excretion of ION904

    Time frame: Up to 24 hours

07

Study locations

1 site
  • BioPharma Services, Inc.
    Toronto, Ontario M9L 3A2, Canada
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 9, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT04731623
Lead sponsor
Ionis Pharmaceuticals, Inc.
Responsible party
Sponsor
First posted
Feb 1, 2021
Start date
Feb 26, 2021
Primary completion
Aug 12, 2021
Completion
Sep 11, 2021
Last update
Aug 9, 2022

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Aug 2022. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Start the discussion