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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03165864




Registration number
NCT03165864
Ethics application status
Date submitted
18/05/2017
Date registered
24/05/2017
Date last updated
22/01/2019

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
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)
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)
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)
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)
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)
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
Sex
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
9/05/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
25/09/2018
Sample size
Target
Accrual to date
Final
36
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/ct2/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

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03165864