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

Phase 1

Completed

• Conditions: Thalassemia
• Interventions: Other: Placebo; Drug: IONIS TMPRSS6-Lrx

• Registration Number: NCT03165864
• Lead Sponsor: Ionis Pharmaceuticals, Inc.

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study Start: 2017-05-09
• Study First Submitted: 2017-05-18
• Study First Submitted QC: 2017-05-22
• Study First Posted: 2017-05-24
• Primary Completion: 2018-09-25
• Study Completion: 2018-09-25
• Status Verified: 2019-01
• Last Update Submitted: 2019-01-17
• Last Update Posted: 2019-01-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 36

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Saline .9%
IONIS TMPRSS6-Lrx	IONIS TMPRSS6-Lrx	Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously

Primary Outcome Measures

Name	Time	Method
Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx	Up to 148 Days	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

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)	Up to 148 Days	The plasma pharmacokinetics (maximum observed drug concentration or Cmax) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)	Up to 148 Days	The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2λz) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)	Up to 148 Days	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
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)	Up to 148 Days	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
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted)	Up to 148 Days	The plasma pharmacokinetics (Percentage of the administered dose excreted in urine (% Dose Excreted) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration

Trial Locations

Locations (1)

Nucleus Network; 🇦🇺 Melbourne, Victoria, Australia