Familial Chylomicronemia Syndrome (FCS) MedDRA version: 20.0 Level: PT Classification code 10059183 Term: Familial hypertriglyceridaemia System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1 Level: LLT Classification code 10020607 Term: Hyperchylomicronemia System Organ Class: 10027433 - Metabolism and nutrition disorders
Conditions
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 2. Satisfactory completion of the ISIS 678354 CS3 index study (last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement 3. Willing to follow a diet comprising = 20 g fat per day during the study Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 55 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5
Exclusion criteria
Exclusion criteria: Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the effect of ISIS 678354 on the percent change in fasting triglycerides (TG) from Baseline;Secondary Objective: To evaluate effect of ISIS 678354 as compared to baseline on: • Percent change in fasting TG upon extended treatment (durability of the effect) • Proportion of patients who achieve = 40% reduction in fasting TG from Baseline • Percent change in fasting apolipoprotein C-III (apoC-III), apolipoprotein B (apoB) and apolipoprotein 48 (apoB48), non-High Density Lipoprotein Cholesterol (non-HDL-C) from Baseline • Proportion of patients who achieve fasting TG = 880 mg/dL • Adjudicated acute pancreatitis event rate during the Treatment Period in patients with = 2 events of adjudicated acute pancreatitis in 5 years prior to treatment with study drug in the index study • Adjudicated acute pancreatitis event rate during the Treatment Period and in patients with prior history of pancreatitis within 10 years prior to screening in index study • Proportion of patients who achieve = 70% reduction in fasting TG from Baseline • Proportion of patients who achieve fasting TG = 500 mg/dL;Primary end point(s): The primary objective is to evaluate safety and tolerability of treatment with ISIS 678354;Timepoint(s) of evaluation of this end point: 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Percent change in fasting TG from Baseline at Month 6 (average of Weeks 23, 25 and 27) • Percent change in fasting TG from Baseline at Month 12 (average of Week 51 and 53), Month 24 (average of Week 103 and Week 105) and Month 36 (average of Week 155 and Week 157) • Proportion of patients who achieve = 40% reduction in fasting TG from Baseline at Month 6 • Proportion of patients who achieve = 40% reduction in fasting TG from Baseline at Month 12, 24, 36 • Percent change in fasting apoC-III from Baseline at Month 6 • Percent change in fasting apoC-III from Month 12, 24, 36 • Percent change in fasting apoB48 from Baseline at Month 6 • Percent change in fasting apoB48 from Baseline at Month 12, 24, 36 • Percent change in fasting non-HDL-C from Baseline at Month 6 • Percent change in fasting non-HDL-C from Baseline at Month 12, 24, 36 • Proportion of patients who achieve fasting TG = 880 mg/dL at Month 6 • Proportion of patients who achieve fasting TG = 880 mg/dL at Month 12, 24, 36 • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 and 157), in patients with = 2 events of adjudicated acute pancreatitis in 5 years prior to treatment with study drug in the index study • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 or 157) • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Weeks 53, 105, or 157) in patients with a prior history of pancreatitis within 10 years prior to Screening in the index study • Proportion of patients who achieve = 70% reduction in fasting TG from Baseline at Month 6 • Proportion of patients who achieve = 70% reduction in fasting TG from Baseline at Month 12, 24, 36 • Proportion of patients who achieve fasting TG = 500 mg/dL at Month 6 • Proportion of patients who achieve fasting TG = 500 mg/dL at Month 12, 24, 36;Timepoint(s) of evaluation of this end point: 6, 12, 24 and 36 months | — |
Countries
Canada, France, Italy, Netherlands, Norway, Portugal, Slovakia, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactStacy Woeppel
Ionis Pharmaceuticals
Outcome results
None listed