Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
NCT ID: NCT02709850
Last Updated: 2020-11-19
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
48 participants
INTERVENTIONAL
2015-11-30
2017-06-26
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
OTHER
DOUBLE
Study Groups
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Cohorts A, D: Placebo
Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1.
Placebo
0.9%NaCl, water, riboflavin
Cohorts A, D: IONIS ANGPTL3-LRx 20 mg
Participants received a single-dose of IONIS ANGPTL3-LRx 20 milligrams (mg) subcutaneously on Day 1.
IONIS ANGPTL3-LRx
Cohorts A, D: IONIS ANGPTL3-LRx 120 mg
Participants received a single-dose of IONIS ANGPTL3-LRx 120 mg subcutaneously on Day 1.
IONIS ANGPTL3-LRx
Cohorts B, C: Placebo
Participants received a single-dose of IONIS ANGPTL3-LRx-matching placebo subcutaneously on Day 1.
Placebo
0.9%NaCl, water, riboflavin
Cohorts B, C: IONIS ANGPTL3-LRx 40 mg
Participants received a single-dose of IONIS ANGPTL3-LRx 40 mg subcutaneously on Day 1.
IONIS ANGPTL3-LRx
Cohorts B, C: IONIS ANGPTL3-LRx 80 mg
Participants received a single-dose of IONIS ANGPTL3-LRx 80 mg subcutaneously on Day 1.
IONIS ANGPTL3-LRx
Cohorts AA-DD: Placebo
Participants received IONIS ANGPTL3-LRx-matching placebo subcutaneously once per week for 6 weeks.
Placebo
0.9%NaCl, water, riboflavin
Cohorts AA-DD: IONIS ANGPTL3-LRx 10 mg
Participants received IONIS ANGPTL3-LRx 10 mg subcutaneously once per week for 6 weeks.
IONIS ANGPTL3-LRx
Cohorts AA-DD: IONIS ANGPTL3-LRx 20 mg
Participants received IONIS ANGPTL3-LRx 20 mg subcutaneously once per week for 6 weeks.
IONIS ANGPTL3-LRx
Cohorts AA-DD: IONIS ANGPTL3-LRx 40 mg
Participants received IONIS ANGPTL3-LRx 40 mg subcutaneously once per week for 6 weeks.
IONIS ANGPTL3-LRx
Cohorts AA-DD: IONIS ANGPTL3-LRx 60 mg
Participants received IONIS ANGPTL3-LRx 60 mg subcutaneously once per week for 6 weeks.
IONIS ANGPTL3-LRx
Interventions
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IONIS ANGPTL3-LRx
Placebo
0.9%NaCl, water, riboflavin
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Males or females 18 to 65 years, inclusive, at the time of informed consent
* Body Mass Index (BMI) ≤ 35.0 kg/m2
* Females must be non-pregnant and non-lactating, and either surgically sterile or postmenopausal.
* Males must be surgically sterile, abstinent or using an acceptable contraceptive method
* Fasting triglycerides (TG) ≥ 150 mg/dL at Screening
* Fasting low density lipoprotein cholesterol (LDL-C) \> 70 mg/dL at Screening
* Fasting TG 90 - 150 mg/dL at Screening
* Fasting LDL-C \> 70 mg/dL at Screening
* Homozygous FH diagnosis and fasting LDL-C ≥ 190 mg/dL (4.9 mmol/L)
* Heterozygous FH diagnosis and fasting LDL-C ≥ 160 mg/dL (4.1 mmol/L)
* Maximally tolerated stable LDL-C lowering agents (stable for at least 12 weeks)
* On stable low-fat diet
* Stable weight (± 4 kg) for ≥ 6 weeks prior to screening
Exclusion Criteria
* Treatment with another Study Drug, biological agent, or device within one-month or 5-half-lives of screening
* Regular use of alcohol within 6 months of screening
* Use of concomitant drugs unless authorized by the Sponsor Medical Monitor
* Known contraindication and/or allergy to heparin
* Smoking \> 10 cigarettes a day
* Considered unsuitable for inclusion by the Principal Investigator
* Myocardial infarction, percutaneous transluminal coronary intervention, or coronary artery bypass graft surgery within 12 weeks prior to screening, or cerebrovascular accident within 24 weeks prior to screening. Participants with adequately treated stable angina, per Investigator assessment, may be included
* Congestive heart failure defined by NYHA Classes III or IV
* Type 2 diabetes mellitus (T2DM) with HbA1c \> 8.0%
* Prior treatment with gene therapy
* Currently receiving apheresis treatments or last apheresis treatment was within 8 weeks of screening
18 Years
65 Years
ALL
Yes
Sponsors
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Akcea Therapeutics
INDUSTRY
Ionis Pharmaceuticals, Inc.
INDUSTRY
Responsible Party
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Locations
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Clinical Site
Toronto, Ontario, Canada
Countries
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References
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Graham MJ, Lee RG, Brandt TA, Tai LJ, Fu W, Peralta R, Yu R, Hurh E, Paz E, McEvoy BW, Baker BF, Pham NC, Digenio A, Hughes SG, Geary RS, Witztum JL, Crooke RM, Tsimikas S. Cardiovascular and Metabolic Effects of ANGPTL3 Antisense Oligonucleotides. N Engl J Med. 2017 Jul 20;377(3):222-232. doi: 10.1056/NEJMoa1701329. Epub 2017 May 24.
Other Identifiers
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2015-004003-23
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ISIS 703802-CS1
Identifier Type: -
Identifier Source: org_study_id