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: 100000004861
Conditions
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • A diagnosis of genetically confirmed Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) • Fasting TG = 880 mg/dL (10 mmol/L) at Screening • History of pancreatitis. Patients without a documented history of pancreatitis are also eligible but their enrollment will be capped at 35% 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: • Acute coronary syndrome within 6 months of Screening • Major surgery within 3 months of Screening • Active pancreatitis within 4 weeks of Screening • Have any other conditions, which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of ISIS 678354 as compared to placebo on the percent change in fasting triglycerides (TG) from Baseline;Secondary Objective: •Proportion of patients who achieve = 40% reduction in fasting TG from Baseline •Percent change in fasting apoB-48 from Baseline •Proportion of patients who achieve fasting TG = 750 mg/dL (8.4 mmol/L) •Adjudicated acute pancreatitis event rate in patients with = 2 events of adjudicated acute pancreatitis in 5 years prior to enrollment •Adjudicated acute pancreatitis event rate •Proportion of patients who achieve = 70% reduction in fasting TG from Baseline •Proportion of patients who achieve fasting TG = 500 mg/dL (5.7 mmol/L) ;Primary end point(s): The primary endpoint is the percent change in fasting TG from Baseline at 6 months (average of Weeks 23, 25 and 27) compared to placebo;Timepoint(s) of evaluation of this end point: 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints include the following: • Percent change in fasting TG from Baseline at 12 months (average of Week 51 and Week 53) compared to placebo • Proportion of patients who achieve = 40% reduction in fasting TG from baseline at 6 months compared to placebo • Percent change in fasting apoB-48 from Bbaseline at 6 months compared to placebo • Proportion of patients who achieve fasting TG = 750 mg/dL at 6 months compared to placebo • Proportion of patients who achieve fasting TG = 500 mg/dL at 6 months compared to placebo • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53) compared to placebo, in patients with = 2 events of adjudicated acute pancreatitis in 5 years prior to enrollment • Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53) compared to placebo • Proportion of patients who achieve = 70% reduction in fasting TG from Baseline at 6 months compared to placebo • Proportion of patients who achieve fasting TG = 500 mg/dL at 6 months compared to placebo ;Timepoint(s) of evaluation of this end point: 6 months and 12 months | — |
Countries
Canada, France, Germany, Hungary, Israel, Italy, Netherlands, Norway, Portugal, Slovakia, Spain, Sweden, United Kingdom, United States
Contacts
Public ContactStacy Woeppel
Ionis Pharmaceuticals
Outcome results
None listed