A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome

JRCT ID: jRCT2031220249

Registered date:07/08/2022

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Familial Chylomicronemia Syndrome (FCS)
• Date of first enrollment: 07/08/2022
• Target sample size: 12
• Countries of recruitment: United States,Japan,United Kingdom,Japan,Ukraina,Japan,Turkey,Japan,Spain,Japan,South Korea,Japan,Singapore,Japan,Serbia,Japan,Russia,Japan,Poland,Japan,New Zealand,Japan,Mexico,Japan,Ireland,Japan,India,Japan,Germany,Japan,France,Japan,Canada,Japan,Belgium,Japan,Belarus,Japan,Austria,Japan,Australia,Japan,Argentina,Japan
• Study type: Interventional
• Intervention(s): ARO-APOC3 (25 mg or 50 mg) or placebo on Day 1, then Q3M for a total of 4 doses.

Outcome(s)

• Primary Outcome: Percent change from baseline at Month 10 in fasting TG

Secondary Outcome

1. Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10
2. Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
3. Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10
4. Percent Change from Baseline in Fasting TG at Month 12
5. Percent Change from Baseline in Fasting APOC3 at Month 12
6. Percent Change from Baseline in Fasting Non-HDL-C at Month 12
7. Percent Change from Baseline in Fasting HDL-C at Month 12
8. Proportion of Patients Achieving TG of < 500 mg/dL at Month 10
9. Proportion of Patients Achieving TG of < 500 mg/dL at Month 12
10. Change from Baseline in Fasting TG Over Time
11. Percent Change from Baseline in Fasting TG Over Time
12. Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
13. Number of Participants with Positively Adjudicated Events of Acute Pancreatitis

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Fasting TG >=10 mmol/L (~880 mg/dL) at screening, that are refractory to standard lipid lowering therapy
2. A diagnosis of FCS
3. Willing to follow dietary counseling as per PI judgment based on local standard of care
4. Participants of childbearing potential (male and female) must use highly effective contraception during the study and for at least 24 weeks following the last dose of study medication. Men must not donate sperm during the study and for at least 24 weeks after the last dose of IP.
5. Women of childbearing potential must have a negative pregnancy test at Screening and cannot be breastfeeding
6. Women of childbearing potential on hormonal contraceptives must be stable on the medication for >=2 menstrual cycles prior to Day 1.

Exclude criteria

1. Current use or use within the last 365 days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
2. Diabetes mellitus with any of the following:
a. Newly diagnosed within 12 weeks of screening
b. HbA1c >=9.0% at screening
-Active pancreatitis within 12 weeks before Day 1
-History of acute coronary syndrome event within 24 weeks of Day 1
-History of major surgery within 12 weeks of Day 1
-Any of the following laboratory values at screening:
a.Uncontrolled hypertension
b.Known history of HIV infection
c.Seropositive for hepatitis B virus (HBV) or hepatitis C virus.
d.New York Heart Association (NYHA) Class II, III, or IV heart failure

Related Information

• Primary Sponsor: Hokonohara Toshihiro