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.
Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)Primary· Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-550.00
± 431.471
Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)Primary· Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-32.83
± 31.994
Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)Secondary· Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-31.983
± 5.4773
Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)Secondary· Baseline to Month 3
Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-51.249
± 4.9093
Fasting Lipid and Lipoprotein Measurements at Month 3Secondary· Month 3
Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Non-HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
217.33
± 119.618
ApoB
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
64.53
± 4.899
HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
13.0
± 1.00
ApoA-1
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
77.3
± 6.03
VLDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
190.17
± 128.861
LDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
27.17
± 10.324
Absolute Change From Baseline to Month 3 in Other Fasting Lipid ParametersSecondary· Baseline to Month 3
Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
Absolute Change From Baseline to Month 3 in TC
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-86.17
± 48.581
Absolute Change From Baseline to Month 3:Non-HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-83.83
± 48.235
Absolute Change From Baseline to Month 3 in ApoB
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-8.12
± 5.352
Absolute Change From Baseline to Month 3 in HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-2.3
± 0.58
Absolute Change From Baseline to Month 3 in ApoA-1
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-17.3
± 11.93
Absolute Change From Baseline to Month 3 in VLDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-82.50
± 51.215
Absolute Change From Baseline to Month 3 in LDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-1.33
± 3.753
Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid ParametersSecondary· Baseline to Month 3
Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.
% Change From Baseline to Month 3 in TC
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-30.39
± 20.436
% Change From Baseline to Month 3 in Non-HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-31.49
± 21.832
% Change From Baseline to Month 3 in ApoB
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-10.73
± 6.021
% Change From Baseline to Month 3 in HDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-15.2
± 3.22
% Change From Baseline to Month 3 in ApoA-1
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-17.8
± 10.97
% Change From Baseline to Month 3 in VLDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-36.03
± 28.044
% Change From Baseline to Month 3 in LDL-C
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-6.47
± 13.426
Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)Secondary· Baseline to Day 92
Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
-338.0
± 38.22
Number of Participants Who Experienced Abdominal Pain During the Treatment PeriodSecondary· Days 1, 29, 57 and 92
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
1
Number of Participants With Treatment Emergent Adverse Events (TEAEs)Secondary· From time of informed consent to end of follow-up period (Up to Week 26)
An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically-significant abnormal laboratory finding, for example), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE is considered related to the investigational drug product. A TEAE was defined as any AE starting on or after the first dose of the study drug.
Group
Value
95% CI
AKCEA-ANGPTL3-LRx 20 mg
3
Adverse events — posted to ClinicalTrials.gov
Time frame: From time of informed consent to end of follow-up period (Up to Week 26)..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03514420 — Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
· Phase 2
· completed
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: 7 January 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/NCT03360747.