A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547

NCT04934891 | Completed Phase 1 FDA Drug

• 1 other identifier: ION547-CS1
• Study Type: interventional
• Target: 48
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the safety and tolerability of single and multiple doses of the antisense inhibitor ION547 administered subcutaneously (SC) in healthy participants.

Conditions

Healthy Volunteers

Keywords

Healthy Participants

Outcome Measures

Primary Outcomes (7)

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

  Up to Day 232

• Cmax: Maximum Observed Plasma Concentration of ION547

  Up to Day 232

• Tmax: Time Taken to Reach Maximal Plasma Concentration of ION547

  Up to Day 232

• AUCt: Area Under the Plasma Concentration-time Curve From Time Zero to Time t for ION547

  Up to Day 232

• Urine 0-24 Hour (hr) Excretion of ION547

  Up to 24 hours

• Change from Baseline in FXII Antigen Levels

  Up to Day 232

• Change from Baseline in FXII Activity Levels

  Up to Day 232

Study Arms (2)

ION547

EXPERIMENTAL

Ascending single multiple doses of ION547 will be administered by SC injection.

Drug: ION547

Placebo

PLACEBO COMPARATOR

Ascending single multiple doses of ION547-matching placebo will be administered by SC injection.

Drug: Placebo

Interventions

ION547 DRUG

ION547 will be administered by SC injection.

ION547

Placebo DRUG

ION547-matching placebo 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 have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
• Healthy males or females 18 to 65, inclusive, at the time of informed consent.
• Satisfy the following:
• a. Females: Women of childbearing potential are not allowed to participate in this study.
• b. Males must be abstinent, surgically sterile (vasectomy with negative semen analysis at Follow-up, or a surgically sterile non-pregnant female partner) or if engaged in sexual relations with a woman of childbearing potential (WOCBP), participant is utilizing a highly effective contraceptive method
• Body mass index (BMI) ≤ 35 kilograms per square meter (kg/m\^2).

You may not qualify if:

• Clinically significant abnormalities in medical history or physical examination.
• Urine protein/creatinine (P/C) ratio greater than or equal to (≥) 0.2 milligrams (mg)/mg.
• Positive test (including trace) for blood on urinalysis.
• Alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, alkaline phosphatase (ALP), serum creatinine, and blood urea nitrogen (BUN) \> upper limit of normal (ULN).
• Hemoglobin A1c (HbA1c) \> ULN
• Platelet count \< lower limit of normal (LLN)
• aPTT \> ULN
• INR \> 1.4
• Evidence of uncorrected hypothyroidism or hyperthyroidism based on thyroid function results at Screening.
• Uncontrolled hypertension (blood pressure \[BP\] \> 160/100 millimeters of mercury \[mmHg\]).
• Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
• Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
• History of bleeding diathesis or coagulopathy.
• Regular use of alcohol within 6 months prior to screening (\> 7 drinks/week for females, \> 14 drinks/week for males \[1 drink = 5 ounces (150 mL) of wine or 12 ounces (360 mL) of beer or 1.5 ounces (45 mL) of hard liquor\]), or use of soft drugs (such as marijuana) within 3 months prior to screening, or hard drugs (such as cocaine and phencyclidine \[PCP\]) within 1 year prior to screening, or positive urine drug screen at Screening.
• Concomitant medication restrictions: the use of prescription and over-the-counter medications including nonsteroidal anti-inflammatory drugs (with the exception of occasional acetaminophen or ibuprofen) and herbal remedies at Screening and throughout the Study Period.

Sponsors & Collaborators

• Ionis Pharmaceuticals, Inc.: lead

Study Sites (1)

BioPharma Services, Inc.

Toronto, Ontario, M9L 3A2, Canada

Location

Study Design

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

Study Record Dates

• First Submitted: June 8, 2021
• First Posted: June 22, 2021
• Study Start: March 24, 2021
• Primary Completion: September 14, 2022
• Study Completion: September 14, 2022
• Last Updated: September 23, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)