CompletedPhase 1/2

Evaluate the Safety and Tolerability, as Well as the Pharmacokinetic and Pharmacodynamic Profiles of Single and Multiple Doses of Eplontersen Administered Subcutaneously to Healthy Volunteers and Patients With Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).

Canada47 participantsStarted 2018-12-21

Plain-language summary

To evaluate the safety and tolerability, as well as the pharmacokinetic and pharmacodynamic profiles of single and multiple doses of Eplontersen administered subcutaneously to healthy volunteers and patients with Hereditary Transthyretin-Mediated Amyloidosis (hATTR ).

Who can participate

Age range18 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal
✓. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method
✓. Weight ≥ 50 kg and BMI \< 32 kg/m\^2

Exclusion criteria

✕. Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion including abnormal safety labs
✕. Drug or alcohol dependency or abuse
✕. Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer
✕. Blood donation within 28 days
✕. Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
✕. Aged 18 to 82 years at the time of informed consent
✕. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal
✕. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method

What they're measuring

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

Timeframe: Up to Day 176

Percentage of Participants Using Concomitant Medications

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Laboratory Values

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Physical Examination Findings

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Electrocardiogram (ECG) Values

Timeframe: Up to Day 176

Trial details

NCT IDNCT03728634

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-02-20

Results submitted2022-10-12

View on ClinicalTrials.gov More Healthy Volunteers trials

Who can participate

Age range18 Years – 65 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal
✓. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method
✓. Weight ≥ 50 kg and BMI \< 32 kg/m\^2

Exclusion criteria

✕. Clinically-significant (CS) abnormalities in medical history, screening laboratory results, physical or physical examination that would render a subject unsuitable for inclusion including abnormal safety labs
✕. Drug or alcohol dependency or abuse
✕. Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer
✕. Blood donation within 28 days
✕. Have any other conditions, which, in the opinion of the Investigator or Sponsor would make the subject unsuitable for inclusion, or could interfere with the subject participating in or completing the Study
✕. Aged 18 to 82 years at the time of informed consent
✕. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal
✕. Males must be surgically sterile or, abstinent or, if engaged in sexual relations with a woman of child-bearing potential, the subject or the subject's non-pregnant female partner must be using a highly effective contraceptive method

What they're measuring

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

Timeframe: Up to Day 176

Percentage of Participants Using Concomitant Medications

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Laboratory Values

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Physical Examination Findings

Timeframe: Up to Day 176

Number of Participants With Clinically Significant Electrocardiogram (ECG) Values

Timeframe: Up to Day 176