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

Healthy Volunteers Clinical Trial

Official title:

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending, Dose-Escalation, Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904 Administered Subcutaneously to Healthy Participants

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04731623
• Other study ID #: ION904-CS1
• Status: Completed
• Phase: Phase 1
• Start date: February 26, 2021
• Est. completion date: September 11, 2021

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: September 11, 2021
• Est. primary completion date: August 12, 2021
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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:

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

Related Conditions & MeSH terms

• Healthy Volunteers

Intervention

Drug:
• ION904
ION904 will be administered by SC injection.
• Placebo
Placebo (0.9% sterile saline) will be administered by SC injection.

Locations

Country	Name	City	State
Canada	BioPharma Services, Inc.	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	Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE)		Up to Day 85
Primary	Severity and dose relationship of TEAE		Up to Day 85
Primary	Severity and dose relationship of clinically significant changes in laboratory values		Up to Day 85
Primary	Severity and dose relationship of clinically significant changes in ECG		Up to Day 85
Secondary	Changes in the Level of ION904 Target Biomarker		Up to Day 85
Secondary	Cmax: Maximum Observed Plasma Concentration of ION904		Up to Day 85
Secondary	Tmax: Time to Reach the Maximum Plasma Concentration of ION904		Up to Day 85
Secondary	t½?z: Plasma Elimination Half-life of ION904		Up to Day 85
Secondary	AUC0-t: Area Under the Plasma Concentration-Time Curve from Time Zero to Time t for ION904		Up to Day 85
Secondary	Urine 0-24 Hour (hr) Excretion of ION904		Up to 24 hours