Clinical trial NCT03360747: Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
Official title: A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRx (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Familial Chylomicronemia Syndrome
Lipoprotein Lipase Deficiency
Hyperlipoproteinemia Type 1
Keywords: Familial Lipoprotein Lipase Deficiency
Hyperlipoproteinemias
Familial Hyperlipoproteinemia Type 1
Hyperlipoproteinemia Type I
Hyperchylomicronemia, Familial
Lipoprotein Lipase Deficiency, Familial
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lead sponsor: Akcea Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Ionis Pharmaceuticals, Inc.
First posted: 2017-12-04
Enrollment: 3
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Primary outcomes:
measure: Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
timeFrame: Baseline to Month 3
measure: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
timeFrame: Baseline to Month 3
Secondary outcomes:
measure: Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)
timeFrame: Baseline to Month 3
measure: Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)
timeFrame: Baseline to Month 3
measure: Fasting Lipid and Lipoprotein Measurements at Month 3
timeFrame: Month 3
measure: Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters
timeFrame: Baseline to Month 3
measure: Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters
timeFrame: Baseline to Month 3
measure: Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)
timeFrame: Baseline to Day 92
measure: Number of Participants Who Experienced Abdominal Pain During the Treatment Period
timeFrame: Days 1, 29, 57 and 92
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
timeFrame: From time of informed consent to end of follow-up period (Up to Week 26)
Other outcomes:
Locations:
city: Montréal
country: Canada
facility: Investigative Site
state: Quebec
Study officials:
References: