[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07727538":3,"trial-entities:NCT07727538":166,"trial-summary:NCT07727538":171},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":10,"sponsor_name":12,"lead_sponsor_class":13,"has_dmc":14,"brief_summary":15,"detailed_description":16,"conditions":17,"keywords":19,"study_type":20,"primary_purpose":21,"phases":22,"enrollment_info":24,"interventions":27,"primary_outcomes":37,"secondary_outcomes":41,"sex":123,"minimum_age":124,"maximum_age":125,"healthy_volunteers":126,"eligibility_criteria":127,"std_ages":131,"locations":133,"central_contacts":158,"overall_officials":163,"references":164,"see_also_links":165},"NCT07727538","ISIS 678354-CS21","A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants","An Open-Label Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Pediatric Patients With Familial Chylomicronemia Syndrome (FCS)","RECRUITING","2031-08","2026-07","2026-07-29","Ionis Pharmaceuticals, Inc.","INDUSTRY",true,"The primary purpose of the study is to evaluate the efficacy of olezarsen administered by subcutaneous injection to pediatric participants with FCS.","This is a Phase 3 multi-center open-label study to evaluate safety, pharmacokinetics (PK), efficacy, and pharmacodynamics (PD) of olezarsen in pediatric participants (aged 2 to less than (\\\u003C)18 years) with FCS. This study consists of three to four periods with the following approximate timeframes: 1-month screening period, 1-year treatment period, an optional 1-year long-term extension treatment period, and a 3-month post-treatment follow-up period.",[18],"Familial Chylomicronemia Syndrome",[],"INTERVENTIONAL","TREATMENT",[23],"PHASE3",{"count":25,"type":26},12,"ESTIMATED",[28],{"type":29,"name":30,"description":31,"armGroupLabels":32,"otherNames":35},"DRUG","Olezarsen","Olezarsen will be administered by subcutaneous injection.",[33,34],"Cohort 1","Cohort 2",[36],"ISIS 678354",[38],{"measure":39,"timeFrame":40},"Percent Change from Baseline in Fasting Triglycerides (TG)","At 6 Months",[42,45,48,50,52,54,56,58,60,62,64,66,68,70,72,74,76,78,80,82,84,86,89,92,95,97,100,102,105,107,110,112,115,117,119,121],{"measure":43,"timeFrame":44},"Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs Including Independently Adjudicated Events of Pancreatitis, and Withdrawals due to Adverse Events (AEs)","Up to 24 Months",{"measure":46,"timeFrame":47},"Change From Baseline in Vital Sign Parameter - Heart Rate (Beats per Minute)","Baseline up to 24 Months",{"measure":49,"timeFrame":47},"Change From Baseline in Vital Sign Parameter - Blood Pressure (Systolic and Diastolic, mmHg)",{"measure":51,"timeFrame":47},"Change From Baseline in Vital Sign Parameter - Oxygen Saturation (%)",{"measure":53,"timeFrame":47},"Change From Baseline in Vital Sign Parameter - Respiratory Rate (Breaths per Minute)",{"measure":55,"timeFrame":47},"Change From Baseline in Vital Sign Parameter - Body Temperature (°C)",{"measure":57,"timeFrame":47},"Change From Baseline in Body Weight (kg)",{"measure":59,"timeFrame":47},"Change From Baseline in Height (cm)",{"measure":61,"timeFrame":47},"Change From Baseline in Pubertal Development Parameter - Sexual Maturity Rating (Tanner Stage)",{"measure":63,"timeFrame":47},"Change From Baseline in Pubertal Development Parameter - Menarche (Age to the Nearest Month and Year)",{"measure":65,"timeFrame":47},"Change From Baseline in Clinical Laboratory Parameter - Amylase",{"measure":67,"timeFrame":47},"Change From Baseline in Clinical Laboratory Parameter - Lipase",{"measure":69,"timeFrame":47},"Change From Baseline in Clinical Laboratory Parameter - Alanine Aminotransferase (ALA)",{"measure":71,"timeFrame":47},"Change From Baseline in Clinical Laboratory Parameter - Aspartate Aminotransferase (AST)",{"measure":73,"timeFrame":47},"Change From Baseline in Clinical Laboratory Parameter - Platelets",{"measure":75,"timeFrame":47},"Change From Baseline in Electrocardiogram (ECG) Parameter - Heart Rate",{"measure":77,"timeFrame":47},"Change From Baseline in ECG Parameters Including PR Interval, QRS Duration, QT Interval, and QT Corrected Interval Fridericia's (QTcF)",{"measure":79,"timeFrame":44},"Peak, Trough (Pre-Dose), and Post-treatment Plasma Concentration of Olezarsen",{"measure":81,"timeFrame":47},"Change From Baseline in Pediatric Quality of Life Inventory (PedsQL) Generic Core Scales",{"measure":83,"timeFrame":47},"Change from Baseline in PedsQL Pediatric Pain Questionnaire (PPQ)",{"measure":85,"timeFrame":47},"Change from Baseline in PedsQL Gastrointestinal Symptoms Module",{"measure":87,"timeFrame":88},"Percent Change from Baseline in Fasting TG","Baseline, 3 Months, 12 Months, 18 Months, 24 Months",{"measure":90,"timeFrame":91},"Proportion of Participants who Achieve Fasting TG \u003C880 milligrams per deciliter (mg\u002FdL)","3 Months, 6 Months, 12 Months, 18 Months, 24 Months",{"measure":93,"timeFrame":94},"Proportion of Participants who Achieve Fasting TG \u003C500 mg\u002FdL","3 Months, 6 Months 12 Months, 18 Months, 24 Months",{"measure":96,"timeFrame":94},"Proportion of Participants who Achieve ≥40 Percent (%) Reduction in Fasting TG from Baseline",{"measure":98,"timeFrame":99},"Percent Change from Baseline in Apolipoprotein C-III (apoC-III), Very Low-density Lipoprotein Cholesterol (VLDL-C), Non High-density Lipoprotein Cholesterol (non-HDL-C)","Baseline, 3 Months, 6 Months, 12 Months, 18 Months, 24 Months",{"measure":101,"timeFrame":99},"Percent Change from Baseline in High-density Lipoprotein Cholesterol (HDL-C) Low-density Lipoprotein Cholesterol (LDL-C), Apolipoprotein B (apoB), Apolipoprotein B-48 (ApoB-48)",{"measure":103,"timeFrame":104},"Event Rate of Adjudicated Acute Pancreatitis","3 Months, 12 Months, 18 Months, 24 Months",{"measure":106,"timeFrame":104},"Percentage of Participants with Abdominal Pain Prior to First Dose vs. TEAEs",{"measure":108,"timeFrame":109},"Change From Baseline in Liver Size as Assessed by Magnetic Resonance Imaging (MRI)","Baseline, 12 Months, 24 Months",{"measure":111,"timeFrame":109},"Change From Baseline in Liver Fat Content as Assessed by MRI",{"measure":113,"timeFrame":114},"Incidence of Anti-Drug Antibodies (ADA) to Olezarsen","Pre-dose on Days 1, 1 Month, 3 Months, 6 Months, 9 Months, 12 Months, 18 Months and 24 Months; and post-dose on Days 1 and 6 Months",{"measure":116,"timeFrame":114},"Titer of ADA to Olezarsen",{"measure":118,"timeFrame":114},"Incidence of Participants with Negative, Treatment-Unaffected, or Treatment-Emergent ADA to Olezarsen",{"measure":120,"timeFrame":114},"Onset of Treatment-Emergent ADA to Olezarsen",{"measure":122,"timeFrame":114},"Time to Onset of Treatment-Emergent ADA to Olezarsen","ALL","2 Years","17 Years",false,{"inclusion":128,"exclusion":129,"raw_text":130},[],[],"Key Inclusion Criteria:\n\n1. Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures.\n2. Must be able to comply with all study procedures.\n3. Age 12 to less than 18 years at the time of informed consent\u002Fassent (Cohort 1); age 2 to less than 12 years at time of informed consent\u002Fassent (Cohort 2).\n4. Willing to fast for at least 10 hours before visits requiring fasted blood sampling.\n5. A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes.\n6. Fasting TGs ≥880 mg\u002FdL at screening. If fasting TG is \\\u003C 880 mg\u002FdL, up to two additional tests may be performed during the screening period with any single test used to qualify.\n\nKey Exclusion Criteria:\n\n1. Diabetes mellitus with any of the following:\n\n   1. Newly diagnosed within 12 weeks prior to screening or during the screening period.\n   2. Hemoglobin A1c (HbA1c) ≥9.5% at screening.\n   3. Change in basal insulin regimen \\>20% within 3 months prior to screening or during the screening period.\n   4. For participants with type 1 diabetes: episode of diabetic ketoacidosis, or ≥3 episodes of severe hypoglycemia within 6 months prior to screening or during the screening period.\n2. History of bleeding, diathesis, or coagulopathy.\n3. Major surgery within 3 months of screening.\n4. Plasma apheresis within 4 weeks prior to screening or planned during the study.\n5. Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer.\n6. Active pancreatitis within 4 weeks prior to screening or during the screening period.\n7. Malignancy diagnosed or treated within 5 years prior to screening or during the screening period.\n\nNote: Other protocol-specified inclusion\u002Fexclusion criteria may apply.",[132],"CHILD",[134,143,151],{"facility":135,"status":8,"city":136,"state":137,"zip":138,"country":139,"geoPoint":140},"UCSF Benioff Children's Hospital","San Francisco","California","94143","United States",{"lat":141,"lon":142},37.77493,-122.41942,{"facility":144,"status":8,"city":145,"state":146,"zip":147,"country":139,"geoPoint":148},"University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"lat":149,"lon":150},32.78306,-96.80667,{"facility":152,"status":8,"city":153,"state":146,"zip":154,"country":139,"geoPoint":155},"Cook Children's Medical Center","Fort Worth","76104",{"lat":156,"lon":157},32.72541,-97.32085,[159],{"name":12,"role":160,"phone":161,"email":162},"CONTACT","(844) 200-6410","IonisOlezarsenPediatric@clinicaltrialmedia.com",[],[],[],{"nct_id":4,"conditions":167,"biomarkers":169},[168],"Familial chylomicronemia syndrome",[170],"FCS-related genes",{"nct_id":4,"found":14,"summary":172,"prompt_version":182},{"design":173,"status":174,"heading":175,"summary":176,"follow_up":177,"word_count":178,"commitments":179,"compensation":180,"drugs_mentioned":181},"This is a Phase 3, multi-center, open-label study, meaning both you and the study team will know you are receiving olezarsen. It plans to enroll about 12 participants.","completed","A Study of Olezarsen for Familial Chylomicronemia Syndrome (FCS) in Children","This study is testing a drug called olezarsen for children aged 2 to 17 years old who have Familial Chylomicronemia Syndrome (FCS). FCS is a rare genetic condition that causes very high levels of fats (triglycerides) in the blood. Olezarsen is given as a shot under the skin. The main goal is to see how much olezarsen can lower fasting triglycerides after 6 months. The study is currently enrolling about 12 participants. To join, a parent or guardian must give permission, and the child must also agree if they are old enough to understand. You must also be able to follow all study procedures.","Participants will be followed for 3 months after their treatment period ends.",104,"The study involves a 1-month screening period, a 1-year treatment period, an optional 1-year extension, and a 3-month follow-up period.","Not stated in the trial record.",[30],"v2"]