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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT03165864




Registration number
NCT03165864
Ethics application status
Date submitted
18/05/2017
Date registered
24/05/2017
Date last updated
21/01/2019

Titles & IDs
Public title
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Scientific 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
Secondary ID [1] 0 0
ISIS 702843-CS1
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thalassemia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - IONIS TMPRSS6-Lrx
Other interventions - Placebo

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

Placebo Comparator: Placebo - Saline .9%


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

Other interventions: Placebo
Saline .9%

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 148 Days
Secondary outcome [1] 0 0
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
Timepoint [1] 0 0
Up to 148 Days
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
Up to 148 Days
Secondary outcome [3] 0 0
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Plasma terminal elimination half-life (t1/2?z) - The plasma pharmacokinetics (Plasma terminal elimination half-life (t1/2?z) of Ionis TMPRSS6-Lrx will be assessed following single and multiple dose SC administration
Timepoint [3] 0 0
Up to 148 Days
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
Up to 148 Days
Secondary outcome [5] 0 0
Pharmacokinetics after single and multiple doses of IONIS TMPRSS6-Lrx (Percentage of the administered dose excreted in urine (% Dose Excreted) - 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
Timepoint [5] 0 0
Up to 148 Days

Eligibility
Key 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
Minimum age
18 Years
Maximum age
65 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key 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
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Nucleus Network - Melbourne
Recruitment postcode(s) [1] 0 0
VIC 3004 - Melbourne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/show/NCT03165864
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications