Phase 2
N=3
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
Familial Chylomicronemia Syndrome · Lipoprotein Lipase Deficiency · Hyperlipoproteinemia Type 1
Bottom Line
View on ClinicalTrials.gov: NCT03360747 ↗Enrolled (actual)
3
Serious AEs
0.0%
Results posted
Jan 2021
Primary outcome: Primary: Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) — -550.00 mg/dL
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AKCEA-ANGPTL3-LRx (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Akcea Therapeutics
- Primary completion
- Jun 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG) |
-550.00 | — |
| PRIMARY Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) |
-32.83 | — |
| SECONDARY Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3) |
-31.983 | — |
| SECONDARY Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3) |
-51.249 | — |
| SECONDARY Fasting Lipid and Lipoprotein Measurements at Month 3 |
217.33; 64.53; 13.0; 77.3; 190.17; 27.17 | — |
| SECONDARY Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters |
-86.17; -83.83; -8.12; -2.3; -17.3; -82.50 | — |
| SECONDARY Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters |
-30.39; -31.49; -10.73; -15.2; -17.8; -36.03 | — |
| SECONDARY Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG) |
-338.0 | — |
| SECONDARY Number of Participants Who Experienced Abdominal Pain During the Treatment Period |
1 | — |
| SECONDARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) |
3 | — |
Summary
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.
Eligibility Criteria
Key Inclusion Criteria
- Genetically confirmed chylomicronemia syndrome.
- Fasting triglycerides greater than or equal to (>=) 750 milligrams per deciliter (mg/dL) [8.4 millimoles per liter (mmol/L)] at Screening.
Key Exclusion Criteria
- Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) >= 9.0%.
- Active pancreatitis within 2 weeks of screening.
- Acute coronary syndrome within 6 months of screening.
- Major surgery within 3 months of screening.
- Treatment with Glybera therapy within 2 years of screening.
- Previous treatment with AKCEA-ANGPTL3-LRx.
- Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.
Data sourced from ClinicalTrials.gov (NCT03360747). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.