CompletedPhase 1ACTRN12616000335493

A Study of Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 696844 Administered Subcutaneously to Healthy Volunteers

A Masked, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of ISIS 696844 Administered Subcutaneously to Healthy Volunteers


Sponsor

Ionis Pharmaceuticals, Inc.

Enrollment

30 participants

Start Date

Jul 5, 2016

Study Type

Interventional

Conditions

Summary

ISIS 696844 is being developed as a treatment for an eye disease called age-related macular degeneration (AMD). This study assesses multiple doses of ISIS 696844 in healthy men and women, to learn about: Levels of drug in the blood over time;Safety and side effects of the drug; Effects of the drug on specific cells and proteins in the body, which will give researchers an idea of how well the drug might work; Which dose levels of the drug may be best to use in future clinical trials. 30 healthy men and women will be divided into 2 equal dose groups, and will each receive a total of 8 doses of study drug (or matching placebo) over a 6 week period. The dose levels planned for the study are: Cohort A: 10 mg study drug or matching placebo; Cohort B: 20 mg study drug or matching placebo; Blood tests to measure drug levels and effects on specific proteins will be taken at certain times during the dosing period, and any changes in health will be recorded. Extra participants may be added to a dose level, or additional dose levels may be studied, depending on ongoing results.


Eligibility

Sex: Both males and femalesMin Age: 25 Yearss

Inclusion Criteria4

  • Must have given written informed consent and be able to comply with study requirements
  • Healthy males or females aged > 25 years 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, if engaged in sexual relations with a female of childbearing potential, the subject must be using an acceptable contraceptive method from the time of signing the informed consent until at least 90 days after the subject’s last dose of Study Drug (ISIS 696844 or placebo)
  • Body weight > 50 kg at the time of informed consent

Exclusion Criteria6

  • Clinical significant abnormalities in medical history, lab tests or physical exam
  • Abnormalities in complement.
  • Known history of or positive test for human immunodeficiency (HIV), hepatitis C or hepatitis B
  • Treatment with another Study Drug, biological agent, or device within one-month of screening
  • Use of concomitant drugs unless authorized by the Sponsor Medical Monitor
  • Smoking > 10 cigarettes a day

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Interventions

Cohort A: 10 mg ISIS 696844 or placebo Cohort B: 20 mg ISIS 696844 or placebo - Subjects will receive 2 subcutaneous doses of Study Drug per week during the first 2 weeks (Days 1, 4, 8, 12) and then

Cohort A: 10 mg ISIS 696844 or placebo Cohort B: 20 mg ISIS 696844 or placebo - Subjects will receive 2 subcutaneous doses of Study Drug per week during the first 2 weeks (Days 1, 4, 8, 12) and then one (1) subcutaneous dose of Study Drug per week for the next 4 weeks (Days 15, 22, 29, 36) for a total of 8 doses.


Locations(2)

Christchurch, New Zealand

Auckland, New Zealand

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ACTRN12616000335493


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