18 and older, any sex, with Familial Chylomicronemia Syndrome or Lipoprotein Lipase Deficiency. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Mean Percent Change From Baseline in Fasting Triglyceride (TG)Primary· Baseline and Months 3, 6, and 12
Baseline for treatment-naïve group was defined as the average of open-label Day 1 pre-dose assessment and the last measurement prior to open-label Day 1. Baseline for CS6-volanesorsen and CS16-volanesorsen arm groups was defined as the average of index study Day 1 pre-dose assessment and the last measurement prior index study Day 1. The values at the Month 3 analysis time point were defined as the average of the Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. The Month 6 analysis time point was at the end of Month 6, and the values were defined as the average of the Week 25 (Day 169
Percent Change at Month 3
Group
Value
95% CI
Treatment-naïve Group
-59.8
± 37.0
CS6-Volanesorsen
-49.2
± 34.8
CS16-Volanesorsen
-64.9
± 9.1
Percent Change at Month 6
Group
Value
95% CI
Treatment-naïve Group
-45.5
± 42.9
CS6-Volanesorsen
-54.8
± 23.8
CS16-Volanesorsen
-43.0
± 19.7
Percent Change at Month 12
Group
Value
95% CI
Treatment-naïve Group
-36.3
± 44.2
CS6-Volanesorsen
-35.1
± 45.6
CS16-Volanesorsen
-41.6
± 36.3
Number of Participants With Treatment-emergent Adverse Events (TEAEs)Primary· From first dose of study drug to end of follow-up period [Up to Week 182]
An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A TEAE was defined as any AE starting or getting worse on or after the first dose of the study drug.
Group
Value
95% CI
Treatment-naïve Group
51
CS6-Volanesorsen
14
CS16-Volanesorsen
3
Adverse events — posted to ClinicalTrials.gov
Time frame: From first dose of study drug to end of follow-up period [Up to Week 182].
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Treatment-naïve Group
Serious: 13/51 (25%)
Deaths: 0/51
CS6-Volanesorsen
Serious: 2/14 (14%)
Deaths: 0/14
CS16-Volanesorsen
Serious: 2/3 (67%)
Deaths: 0/3
Serious adverse events (17 terms)
Reaction
System
Treatment-naïve Group
CS6-Volanesorsen
CS16-Volanesorsen
Thrombocytopenia
Blood and lymphatic system disorders
—
—
—
Pancreatitis
Gastrointestinal disorders
—
—
—
Pancreatitis acute
Gastrointestinal disorders
—
—
—
Pancreatitis chronic
Gastrointestinal disorders
—
—
—
Arthritis
Musculoskeletal and connective tissue disorders
—
—
—
Intervertebral disc protrusion
Musculoskeletal and connective tissue disorders
—
—
—
Myalgia
Musculoskeletal and connective tissue disorders
—
—
—
Tendon calcification
Musculoskeletal and connective tissue disorders
—
—
—
Influenza like illness
General disorders
—
—
—
Diverticulitis
Infections and infestations
—
—
—
Urinary tract infection
Infections and infestations
—
—
—
Clavicle fracture
Injury, poisoning and procedural complications
—
—
—
Haemoglobin decreased
Investigations
—
—
—
Diabetes mellitus inadequate control
Metabolism and nutrition disorders
—
—
—
Abortion spontaneous
Pregnancy, puerperium and perinatal conditions
—
—
—
Focal segmental glomerulosclerosis
Renal and urinary disorders
—
—
—
Ovarian cyst
Reproductive system and breast disorders
—
—
—
Other adverse events (126 terms — click to expand)
NCT02910635 — A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
· Phase 1
· completed
NCT02300233 — The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients With Hypertriglyceridemia
· Phase 3
· completed
NCT03544060 — Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
· no longer available
Other recruiting trials for Familial Chylomicronemia Syndrome
Currently open trials in the same condition.
NCT06471543 — Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects
· Phase 1, PHASE2
· active not recruiting
Other Akcea Therapeutics trials
Trials by the same sponsor.
NCT04850105 — A Non-interventional Cohort Safety Study of Patients With hATTR-PN
· recruiting
NCT04306510 — A Study to Characterize Adverse Events Occurring Within One Day of TEGSEDI Administration to Adult Participants With hAT
· Phase 4
· terminated
NCT03514420 — Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
· Phase 2
· completed
NCT03455777 — Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Homozygous Familial Hypercholesterolemia (HoFH)
· Phase 2
· withdrawn
NCT03506854 — Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
· Phase 1
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Akcea Therapeutics
Last refreshed: 26 August 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02658175.