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Clinical Trials/NCT04998201
NCT04998201
Completed
Phase 2

A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of Plozasiran in Adults With Mixed Dyslipidemia

Arrowhead Pharmaceuticals30 sites in 6 countries353 target enrollmentSeptember 28, 2021

Overview

Phase
Phase 2
Intervention
Placebo
Conditions
Mixed Dyslipidemia
Sponsor
Arrowhead Pharmaceuticals
Enrollment
353
Locations
30
Primary Endpoint
Percent Change From Baseline at Week 24 in Fasting TG
Status
Completed
Last Updated
18 days ago

Overview

Brief Summary

Participants who have met all protocol eligibility criteria will be randomly assigned to treatment (ARO-APOC3 or placebo) in a double-blind fashion and will be evaluated for safety and efficacy over 48 weeks. Participants will be counseled to remain on a specified diet throughout the study, as recommended by the Investigator in accordance with local standards of care. After week 48, participants will be eligible and invited to consent and continue in an open-label extension study. All placebo participants who opt to continue will switch to active drug (ARO-APOC3) during the extension study.

Registry
clinicaltrials.gov
Start Date
September 28, 2021
End Date
August 14, 2023
Last Updated
18 days ago
Study Type
Interventional
Study Design
Parallel
Sex
All

Investigators

Responsible Party
Sponsor

Eligibility Criteria

Inclusion Criteria

  • Based on medical history, prior evidence of triglycerides (TG) ≥ 150 milligrams (mg)/deciliter (dL) and ≤ 499 mg/dL
  • Fasting levels at Screening of non-high-density lipoprotein cholesterol (non-HDL-C) ≥ 100 mg/dL or low-density lipoprotein cholesterol (LDL-C) ≥ 70 mg/dL after at least 2 weeks of stable diet and 4 weeks on stable optimal statin therapy
  • Mean fasting TG ≥ 150 mg/dL and ≤ 499 mg/dL during Screening collected at two separate and consecutive visits and at least 7 days apart and not more than 17 days apart
  • Willing to follow diet counseling as per Investigator judgment based on local standard of care
  • Participants of childbearing potential (males \& females) must use highly effective contraception during the study and for at least 24 weeks following the last dose of study medication. Males must not donate sperm and females must not donate eggs during the study and for at least 24 weeks following the last dose of study medication.
  • Women of childbearing potential must have a negative pregnancy test at Screening and cannot be breastfeeding
  • Women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to Day 1
  • Willing to provide written informed consent and to comply with study requirements

Exclusion Criteria

  • Current use or use within 365 days from Day 1 of any hepatocyte targeted short interfering RNA oligonucleotides (siRNA) or antisense oligonucleotide molecule
  • Active pancreatitis within 12 weeks prior to Day 1
  • Any planned bariatric surgery or similar procedures to induce weight loss from consent through end of study
  • Acute coronary syndrome event within 24 weeks of Day 1
  • History of major surgery within 12 weeks of Day 1 or planned major surgery during the study
  • Planned coronary intervention (such as, stent placement or heart bypass) during the study
  • New York Heart Association (NYHA) Class II, III or IV heart failure or last known ejection fraction of \<30%
  • Uncontrolled hypertension
  • Known history of human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B virus (HBV), seropositive for Hepatitis C virus (HCV)
  • Uncontrolled hypothyroidism or hyperthyroidism

Arms & Interventions

Placebo

Volume-matched placebo SC injection on Day 1 and Week 12 for a total of 2 injections

Intervention: Placebo

ARO-APOC3 50 mg Q24W

ARO-APOC3 50 mg SC injection on Day 1 and Week 24 (Q24W) for a total of 2 injections

Intervention: ARO-APOC3

ARO-APOC3 50 mg Q12W

ARO-APOC3 50 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections

Intervention: ARO-APOC3

ARO-APOC3 10 mg Q12W

ARO-APOC3 10 milligrams (mg) subcutaneous (SC) injection on Day 1 and Week 12 (Q12W) for a total of 2 injections

Intervention: ARO-APOC3

ARO-APOC3 25 mg Q12W

ARO-APOC3 25 mg SC injection on Day 1 and Week 12 (Q12W) for a total of 2 injections

Intervention: ARO-APOC3

Outcomes

Primary Outcomes

Percent Change From Baseline at Week 24 in Fasting TG

Time Frame: Baseline, Week 24

Least square (LS) mean and standard error (SE) were calculated using mixed model repeated measures (MMRM) which included treatment arm, study visit, and baseline value as model terms and treatment by visit and treatment by baseline as interaction terms.

Secondary Outcomes

  • Percent Change From Baseline Over Time Through Week 48 in Fasting TG(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/early termination (ET))
  • Percent Change From Baseline at Week 24 in Apolipoprotein (Apo)C-III(Baseline, Week 24)
  • Percent Change From Baseline Over Time Through Week 48 in ApoC-III(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/ET)
  • Percent Change From Baseline at Week 24 in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C)(Baseline, Week 24)
  • Percent Change From Baseline Over Time Through Week 48 in Fasting Non-HDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/ET)
  • Percent Change From Baseline at Week 24 in Fasting High-Density Lipoprotein Cholesterol (HDL-C)(Baseline, Week 24)
  • Percent Change From Baseline Over Time Through Week 48 in Fasting HDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/ET)
  • Percent Change From Baseline at Week 24 in Fasting Total Apolipoprotein B (ApoB)(Baseline, Week 24)
  • Percent Change From Baseline Over Time Through Week 48 in Fasting Total ApoB(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/ET)
  • Percent Change From Baseline at Week 24 in Fasting Low-Density Lipoprotein-Cholesterol (LDL-C)(Baseline, Week 24)
  • Percent Change From Baseline Over Time Through Week 48 in Fasting LDL-C(Baseline, Weeks 4, 8, 12, 16, 20, 24, 28, 36, 48/ET)
  • Number of Participants With Treatment-Emergent Adverse Events (TEAEs)(Up to Week 48)

Study Sites (30)

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