An Open-Label Extension Study of AKCEA-APOCIII-LRX Administered to Patients with Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia Syndrome (FCS)
- Registration Number
- 2023-509029-29-00
- Lead Sponsor
- Ionis Pharmaceuticals Inc.
- Brief Summary
To evaluate the effect of olezarsen on the percent change in fasting triglycerides (TG) from Baseline
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Ongoing, recruiting
- Sex
- Not specified
- Target Recruitment
- 30
Satisfactory completion of the ISIS 678354 CS3 index study (last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement
Willing to follow a diet comprising ≤ 20 g fat per day during the study
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)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method The primary objective is to evaluate safety and tolerability of treatment with olezarsen The primary objective is to evaluate safety and tolerability of treatment with olezarsen
- Secondary Outcome Measures
Name Time Method Proportion of patients who achieve fasting TG ≤ 500 mg/dL at Month 6, 12, 24, 36, 48 Proportion of patients who achieve fasting TG ≤ 500 mg/dL at Month 6, 12, 24, 36, 48
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 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), and Month 48 (average of Week 205 and 209) 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), and Month 48 (average of Week 205 and 209)
Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline at Month 6, 12, 24, 36, 48 Proportion of patients who achieve ≥ 40% reduction in fasting TG from Baseline at Month 6, 12, 24, 36, 48
Percent change in fasting apoB48 from Baseline at Month 6, 12, 24, 36, 48 Percent change in fasting apoB48 from Baseline at Month 6, 12, 24, 36, 48
Percent change in fasting apoC-III from Baseline at Month 6, 12, 24, 36, 48 Percent change in fasting apoC-III from Baseline at Month 6, 12, 24, 36, 48
Percent change in fasting non-HDL from Baseline at Month 6, 12, 24, 36, 48 Percent change in fasting non-HDL from Baseline at Month 6, 12, 24, 36, 48
Proportion of patients who achieve fasting TG 880mg/dL at Month 6, 12, 24, 36, 48 Proportion of patients who achieve fasting TG 880mg/dL at Month 6, 12, 24, 36, 48
Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105, 157 or 209) Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105, 157 or 209)
Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 157 or 209), 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, 105 157 or 209), 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, 105 157 or 209) in patient with a prior history of pancreatitis within 10 years prior to Screening in the index study Adjudicated acute pancreatitis event rate during the Treatment Period (Week 1 through Week 53, 105 157 or 209) in patient 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, 12, 24, 36, 48 Proportion of patients who achieve ≥ 70% reduction in fasting TG from Baseline at Month 6, 12, 24, 36, 48
Trial Locations
- Locations (14)
Sahlgrenska University Hospital-Vaestra Goetalandsregionen
🇸🇪Goteborg, Sweden
Centre Hospitalier Universitaire De Dijon
🇫🇷Dijon, France
Hospices Civils De Lyon
🇫🇷Bron, France
Centre Hospitalier Regional De Marseille
🇫🇷Marseille, France
Azienda Ospedaliero-Universitaria Policlinico Umberto I
🇮🇹Rome, Italy
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
🇮🇹Palermo, Italy
Azienda Ospedaliera Universitaria Federico II Di Napoli
🇮🇹Naples, Italy
Hospital Universitario 12 De Octubre
🇪🇸Madrid, Spain
Hospital Clinic De Barcelona
🇪🇸Barcelona, Spain
University Hospital Virgen Del Rocio S.L.
🇪🇸Sevilla, Spain
Scroll for more (4 remaining)Sahlgrenska University Hospital-Vaestra Goetalandsregionen🇸🇪Goteborg, SwedenStefano RomeoSite contact46768672634stefano.romeo@wlab.gu.se