Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia
- Registration Number
- NCT05089084
- Lead Sponsor
- Arrowhead Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Active, not recruiting
- Sex
- All
- Target Recruitment
- Not specified
Inclusion Criteria:<br><br> - Fasting TG = 10 mmol/L (= 880 mg/dL) at screening refractory to standard lipid<br> lowering therapy<br><br> - Diagnosis of FCS<br><br> - Willing to follow dietary counseling as per investigator judgement based on local<br> standard of care<br><br> - Participants of childbearing potential (males & females) must use highly-effective<br> contraception during the study and for at least 24 weeks following the last dose of<br> study medication. Males must not donate sperm during the study and for at least 24<br> weeks following the last dose of study medication<br><br> - Women of childbearing potential must have a negative pregnancy test at Screening and<br> cannot be breastfeeding<br><br> - Women of childbearing potential on hormonal contraceptives must be stable on the<br> medication for = 2 menstrual cycles prior to Day 1<br><br>Exclusion Criteria:<br><br> - Current use or use within the last 365 Days from Day 1 of any hepatocyte-targeted<br> siRNA or antisense oligonucleotide molecule<br><br> - Diabetes mellitus newly diagnosed within 12 weeks of Screening or where HbA1c = 9.0%<br> at Screening<br><br> - Active pancreatitis within 12 weeks before Day 1<br><br> - History of acute coronary syndrome event within 24 weeks of Day 1<br><br> - History of major surgery within 12 weeks of Day 1<br><br> - Uncontrolled hypertension<br><br> - On treatment with human immunodeficiency virus (HIV) antiretroviral therapy<br><br> - Seropositive for hepatitis B virus (HBV) or hepatitis C virus (HCV)<br><br> - New York Heart Association (NYHA) Clas II, III, or IV heart failure<br><br>Note: Additional Inclusion/Exclusion criteria may apply per protocol
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10
- Secondary Outcome Measures
Name Time Method Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged);Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10;Percent Change from Baseline in Fasting APOC3 at Month 12;Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10;Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10;Percent Change from Baseline in Fasting TG at Month 12;Percent Change from Baseline in Fasting Non-HDL-C at Month 12;Percent Change from Baseline in Fasting HDL-C at Month 12;Proportion of Patients Achieving TG of < 500 mg/dL at Month 10;Proportion of Patients Achieving TG of < 500 mg/dL at Month 12;Change from Baseline in Fasting TG Over Time;Percent Change from Baseline in Fasting TG Over Time;Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs);Number of Participants with Positively Adjudicated Events of Acute Pancreatitis