Clinical trial NCT03165864: Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
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 ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Thalassemia
Keywords: Thalassaemia, IONIS TMPRSS6-Lrx
Lead sponsor: Ionis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2017-05-24
Enrollment: 36
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


Eligibility criteria: 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

Primary outcomes:
measure: Incidence and severity of adverse events that are related to treatment with IONIS TMPRSS6-Lrx
timeFrame: Up to 148 Days
Secondary outcomes:
measure: Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (maximum observed drug concentration or Cmax)
timeFrame: Up to 148 Days
measure: Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (time taken to reach maximal concentration or Tmax)
timeFrame: Up to 148 Days
measure: Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2λz)
timeFrame: Up to 148 Days
measure: 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)
timeFrame: Up to 148 Days
measure: Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted)
timeFrame: Up to 148 Days
Other outcomes:
measure: Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in transferrin saturation)
timeFrame: Up to 148 Days
measure: Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in serum iron)
timeFrame: Up to 148 Days
measure: Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in hepcidin levels)
timeFrame: Up to 148 Days
Locations:
city: Melbourne
country: Australia
facility: Nucleus Network
state: Victoria
Study officials:
References: