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Phase 2 N=204 Randomized Quadruple-blind Treatment

Study of ARO-ANG3 in Adults With Mixed Dyslipidemia

Mixed Dyslipidemia

Enrolled (actual)
204
Serious AEs
8.6%
Results posted
Jan 2024
Primary outcome: Primary: Percent Change From Baseline in Fasting TG at Week 24 — 7.55; -43.67; -49.01; -55.57 percentage change — p=<.0001

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
ARO-ANG3 (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Arrowhead Pharmaceuticals
Primary completion
Aug 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change From Baseline in Fasting TG at Week 24
7.55; -43.67; -49.01; -55.57 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting TG Over Time
7.00; -41.52; -52.22; -56.21; 13.58; -41.01 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) at Week 24
4.2; -25.1; -24.6; -32.2 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Non-HDL-C Over Time
5.2; -23.5; -25.3; -32.9; 3.7; -24.4 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Total Apolipoprotein B (ApoB) at Week 24
2.27; -16.40; -12.91; -19.64 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Total ApoB Over Time
2.57; -14.00; -11.71; -18.47; 1.51; -15.85 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting Low-density Lipoprotein-Cholesterol (LDL-C) Using Ultracentrifugation at Week 24
5.0; -11.0; -4.3; -15.1 0.0138 sig
SECONDARY
Percent Change From Baseline in Fasting LDL-C Using Ultracentrifugation Over Time
5.1; -8.3; -3.4; -13.7; 3.7; -10.1 0.0192 sig
SECONDARY
Percent Change From Baseline in Angiopoietin-like Protein 3 (ANGPTL3) at Week 24
1.24; -53.02; -68.52; -72.45 <.0001 sig
SECONDARY
Percent Change From Baseline in ANGPTL3 Over Time
5.54; -62.11; -75.25; -79.15; 2.97; -56.09 <.0001 sig
SECONDARY
Percent Change From Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) at Week 24
3.1; -9.0; -18.5; -21.5 0.0039 sig
SECONDARY
Percent Change From Baseline in Fasting HDL-C Over Time
-0.3; -13.9; -25.5; -24.8; 0.4; -14.5 0.0003 sig
SECONDARY
Plasma Pharmacokinetic (PK) Concentration for ARO-ANG3 Over Time in the Double-Blind Treatment Period
0.0000; 2.6685; 0.0000; 57.6941; 63.5267; 154.7823
SECONDARY
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and/or Serious TEAEs up to Week 24
30; 37; 29; 39; 3; 3
SECONDARY
Number of Participants With TEAEs and/or SAEs Over Time in the Double-Blind Treatment Period
35; 40; 34; 42; 8; 12
SECONDARY
Number of Participants With AEs and/or SAEs Over Time in the Open-Label Extension (OLE) Period
10; 27; 13; 30; 10; 31
SECONDARY
Percent Change From Baseline in Fasting TG Over Time in the Open Label Extension (OLE) Period
-10.02; -34.78; -2.09; -39.64; 7.87; -51.88
SECONDARY
Percent Change From Baseline in Fasting Non-HDL-C Over Time in the Open Label Extension (OLE) Period
6.70; -19.95; -5.02; -16.02; -2.53; -23.12
SECONDARY
Percent Change From Baseline in Fasting Total ApoB Over Time in the Open Label Extension (OLE) Period
6.57; -13.49; -10.22; -7.48; -3.88; -11.77
SECONDARY
Percent Change From Baseline in Fasting LDL-C Using Ultracentrifugation Over Time in the Open Label Extension (OLE) Period
16.61; -7.68; -1.59; 4.59; 4.13; -2.82
SECONDARY
Percent Change From Baseline in ANGPTL3 Over Time in the Open Label Extension (OLE) Period
3.41; -41.57; 4.47; -56.81; 0.79; -59.24
SECONDARY
Percent Change From Baseline in Fasting HDL-C Over Time in the Open Label Extension (OLE) Period
13.70; 1.67; 7.70; -12.88; 4.80; -11.26

Summary

The purpose of AROANG3-2001 is to evaluate the efficacy and safety of ARO-ANG3 in participants with mixed dyslipidemia. Participants will initially receive 2 subcutaneous injections of ARO-ANG3 or placebo. Participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of ARO-ANG3.

Eligibility Criteria

Inclusion Criteria

  • Based on medical history, evidence of triglycerides (TG) ≥ 150 mg/dL but ≤ 499 mg/dL
  • Fasting levels at Screening of LDL-C ≥ 70 mg/dL OR non-HDL-C ≥ 100 mg/dL after at least 4 weeks of stable diet and 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 and maintain a stable diet per Investigator judgment based on local standard of care
  • Participants of childbearing potential must agree to use highly-effective contraception during the study and for at least 24 weeks from last dose of study medication
  • Women of childbearing potential must have a negative pregnancy test 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
  • Men must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
  • Able and 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 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 to end of study
  • Acute coronary syndrome event within 24 weeks of Day 1
  • Major surgery within 12 weeks of Day 1 or planned surgery during the study
  • Planned coronary intervention (e.g., stent placement or heart bypass) during the study
  • Uncontrolled hypertension
  • Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B (HBV), seropositive for Hepatitis C (HCV)
  • Uncontrolled hypothyroidism or hyperthyroidism
  • Hemorrhagic stroke within 24 weeks of Day 1
  • History of bleeding diathesis or coagulopathy
  • Current diagnosis of nephrotic syndrome
  • Systemic use of corticosteroids or anabolic steroids within 4 weeks prior to Day 1 or planned use during the study
  • Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply)

Note: additional inclusion/exclusion criteria may apply per protocol

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04832971). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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