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

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ClinicalTrials.gov Identifier: NCT04934891
Recruitment Status : Recruiting
First Posted : June 22, 2021
Last Update Posted : October 18, 2021
Sponsor:
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Healthy Volunteers Drug: ION547 Drug: Placebo Phase 1

Detailed Description:
This is a Phase 1, double-blind, randomized, placebo-controlled, dose escalation study of ION547 in up to 76 participants. Participants will be randomized to receive single and multiple doses of ION547 or placebo SC. The maximum length of participation in the study will be approximately 34 weeks.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 76 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Double-Blind, Placebo-Controlled, Dose-Escalation Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547 Administered Subcutaneously to Healthy Subjects
Actual Study Start Date : May 4, 2021
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : June 2022
Arms and Interventions
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Arm Intervention/treatment
Experimental: ION547
Ascending single multiple doses of ION547 will be administered by SC injection.
 Drug: ION547
ION547 will be administered by SC injection.
Placebo Comparator: Placebo
Ascending single multiple doses of ION547-matching placebo will be administered by SC injection.
 Drug: Placebo
ION547-matching placebo will be administered by SC injection.


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With at Least One Treatment-emergent Adverse Event (TEAE) [ Time Frame: Up to Day 232 ]
  2. Cmax: Maximum Observed Plasma Concentration of ION547 [ Time Frame: Up to Day 232 ]
  3. Tmax: Time Taken to Reach Maximal Plasma Concentration of ION547 [ Time Frame: Up to Day 232 ]
  4. AUCt: Area Under the Plasma Concentration-time Curve From Time Zero to Time t for ION547 [ Time Frame: Up to Day 232 ]
  5. Urine 0-24 Hour (hr) Excretion of ION547 [ Time Frame: Up to 24 hours ]
  6. Change from Baseline in FXII Antigen Levels [ Time Frame: Up to Day 232 ]
  7. Change from Baseline in FXII Activity Levels [ Time Frame: Up to Day 232 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  1. 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.
  2. Healthy males or females 18 to 65, inclusive, at the time of informed consent.

  3. Satisfy the following:

    a. Females: Women of childbearing potential are not allowed to participate in this study.

  4. 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
  5. Body mass index (BMI) ≤ 35 kilograms per square meter (kg/m^2).

Exclusion Criteria:

  1. Clinically significant abnormalities in medical history or physical examination.

  2. Screening laboratory results as follows, or any other clinically significant (CS) abnormalities in screening laboratory values that would render a participant unsuitable for inclusion:

    1. Urine protein/creatinine (P/C) ratio greater than or equal to (≥) 0.2 milligrams (mg)/mg.
    2. Positive test (including trace) for blood on urinalysis.
    3. Alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, alkaline phosphatase (ALP), serum creatinine, and blood urea nitrogen (BUN) > upper limit of normal (ULN).
    4. Hemoglobin A1c (HbA1c) > ULN
    5. Platelet count < lower limit of normal (LLN)
    6. aPTT > ULN
    7. INR > 1.4
    8. Evidence of uncorrected hypothyroidism or hyperthyroidism based on thyroid function results at Screening.
  3. Uncontrolled hypertension (blood pressure [BP] > 160/100 millimeters of mercury [mmHg]).
  4. 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.
  5. Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer.
  6. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid [siRNA]) within 4 months of screening if single dose received, or within 12 months of screening if multiple doses received. Exclusion does not apply to Covid-19 messenger ribonucleic acid (mRNA) vaccines.
  7. History of bleeding diathesis or coagulopathy.
  8. 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.
  9. 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.
  10. Smoking limitations: more than 10 cigarettes/day at Screening and those with a significant change in smoking habits within 1 month prior to Screening should be excluded.
  11. Blood donation of 50 to 499 mL within 30 days of Screening or of > 499 mL within 60 days of Screening.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04934891


Contacts
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Contact: Ionis Pharmaceuticals (844) 414-1645 ionisNCT04934891study@clinicaltrialmedia.com

Locations
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Canada, Ontario
BioPharma Services, Inc. Recruiting
Toronto, Ontario, Canada, M9L 3A2
Contact: https://www.biopharmaservices.com/volunteers/register/         
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04934891    
Other Study ID Numbers: ION547-CS1
First Posted: June 22, 2021    Key Record Dates
Last Update Posted: October 18, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Ionis Pharmaceuticals, Inc.:
Healthy Participants