Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers

Ionis Pharmaceuticals, Inc.36 enrolled

Overview

The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Thalassemia

Interventions

IONIS TMPRSS6-LrxDRUG

Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously

PlaceboOTHER

Saline .9%

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: * Must have given written informed consent and be able to comply with all study requirements * Healthy males or females aged 18-65 inclusive 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, abstinent or using an acceptable contraceptive method * BMI \< 32 kg/m2 Exclusion Criteria: * Clinically significant abnormalities in medical history or physical examination * Clinically significant lab abnormalities that would render a subject unsuitable for inclusion * Known history or positive test for HIV, HCV, or HBV * Treatment with another Study Drug, biological agent, or device within one-month of Screening or 5 half-lives of investigational agent, whichever is longer * Smoking \> 10 cigarettes per day * Regular excessive use of alcohol within 6 months of screening * Current use of concomitant medications other than occasional acetaminophen (paracetamol) or ibuprofen unless approved by Sponsor Medical Monitor * Considered unsuitable for inclusion by the Investigator or Sponsor

Locations (1)

Nucleus Network

Melbourne, Victoria, Australia

Outcomes

Primary Outcomes

Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx

The safety and tolerability of single and multiple doses of IONIS TMPRSS6-Lrx will be assessed by determining the incidence, severity, and dose relationship of adverse events that are related to treatment with IONIS TMPRSS6-Lrx

Time frame: Up to 148 Days

Secondary Outcomes

Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)

The plasma pharmacokinetics (maximum observed drug concentration or Cmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

Time frame: Up to 148 Days

Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)

The plasma pharmacokinetics (time taken to reach maximal concentration or Tmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

Time frame: Up to 148 Days

Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)

The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2λz) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

Time frame: Up to 148 Days

Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt)

The plasma pharmacokinetics (Partial areas under the plasma concentration-time curve from zero time (predose) to selected times (t) after the subcutaneous administration (AUCt) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

Data from ClinicalTrials.gov

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