Thalassemia
Conditions
Keywords
Thalassaemia, IONIS TMPRSS6-Lrx
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
Interventions
DRUGIONIS TMPRSS6-Lrx
Ascending single and multiple doses of IONIS TMPRSS6-Lrx administered subcutaneously
OTHERPlacebo
Saline .9%
Sponsors
Ionis Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 (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 (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 |
| 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 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in hepcidin levels) | Up to 148 Days | Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in hepcidin levels compared to baseline |
| Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in serum iron) | Up to 148 Days | Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in serum iron compared to baseline |
| Pharmacodynamics after single and multiple doses of IONIS TMPRSS6-Lrx (Changes in transferrin saturation) | Up to 148 Days | Effects of IONIS TMPRSS6-Lrx on changes (absolute and percent) in transferrin saturation compared to baseline |
Countries
Australia
Outcome results
None listed