A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia Syndrome
- Interventions
- Registration Number
- NCT05130450
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Brief Summary
The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.
- Detailed Description
This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period.
Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 60
• Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement.
• Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant 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
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Olezarsen Olezarsen Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 153.
- Primary Outcome Measures
Name Time Method Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline Baseline and 6 months
- Secondary Outcome Measures
Name Time Method Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months At 6 months Percentage of Participants Who Achieve Fasting TG ≤ 880 mg/dL at 12, 24, 36 months At 12, 24, 36 months Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months At 6 months Percent Change in Fasting non-HDL-C from Months 12, 24, 36 At 12, 24, 36 months Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Participants With ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Treatment With Study Drug in the Index Study Week 1 through Week 157 Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Week 1 through Weeks 53, 105, or 157 Percent Change in Fasting apoC-III from Baseline at Month 6 Baseline and 6 months Percentage of Participants Who Achieve Fasting TG ≤ 880 milligrams per deciliter (mg/dL) at 6 Months At 6 months Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Patients with a Prior History of Pancreatitis within 10 Years Prior to Screening in the Index Study Week 1 through Weeks 53, 105, or 157 Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months At 12, 24, 36 months Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months At 12, 24, 36 months Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months At 12, 24, 36 months Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months At 6 months Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) at 24 Months (Average of Week 103 and Week 105) and 36 Months (Average of Week 155 and Week 157) Baseline and 36 months Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months At 6 months Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months At 12, 24, 36 months Percent Change in Fasting apoC-III from Month 12, 24, 36 At 12, 24, 36 months Percent Change in Fasting non-HDL-C from Baseline at Month 6 Baseline and 6 months
Trial Locations
- Locations (27)
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
University of Kansas Medical Center (KUMC)
🇺🇸Kansas City, Kansas, United States
IDS Central
🇺🇸Philadelphia, Pennsylvania, United States
Hôpital Bicêtre
🇫🇷Le Kremlin-Bicêtre, France
Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum
🇳🇱Amsterdam, Noor-Holland, Netherlands
Hospital da Senhora da Oliveira Guimaraes
🇵🇹Creixomil, Portugal
Excel Medical Clinical Trials, LLC
🇺🇸Boca Raton, Florida, United States
Via Sergio Pansini 5
🇮🇹Napoli, Italy
Azienda Ospedaliero Universitaria Policlinico Umberto I
🇮🇹Rome, Italy
Clinique des Maladies Lipidiques de Quebec Inc.
🇨🇦Québec, Quebec, Canada
Pharmacie Hopital de la Conception
🇫🇷Marseille, France
UOC di Farmacia AOUP PAOLO GIACCONE
🇮🇹Palermo, Italy
Oslo Hospital Pharmacy Rikshospitalet
🇳🇴Oslo, Norway
Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz
🇵🇹Lisboa, Portugal
Ecogene-21
🇨🇦Chicoutimi, Quebec, Canada
Nathalie Saint-Pierre
🇨🇦Montréal, Quebec, Canada
Diabetes/Lipid Management & Research Center
🇺🇸Huntington Beach, California, United States
Department of Pharmacy
🇺🇸Park Ridge, Illinois, United States
CTSI Investigational Pharmacy
🇺🇸New York, New York, United States
Milstein Hospital
🇺🇸New York, New York, United States
Institute de Recherches Cliniques de Montreal
🇨🇦Montreal, Quebec, Canada
Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique
🇫🇷Bron, France
Metabolicke centrum MU
🇸🇰Bratislava, Slovakia
Hospital Clínic Barcelona C/ Villarroel
🇪🇸Barcelona, Spain
Apokteket AB
🇸🇪Malmö, Sweden
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain
Royal Manchester Children's Hospital
🇬🇧Manchester, United Kingdom