Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03360747
• Other study ID #: ISIS 703802-CS3
• Status: Completed
• Phase: Phase 2
• Start date: December 21, 2017
• Est. completion date: September 4, 2018

Study information

• Verified date: December 2020
• Source: Akcea Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: September 4, 2018
• Est. primary completion date: June 12, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Familial Chylomicronemia Syndrome
• Hyperlipidemias
• Hyperlipoproteinemia Type 1
• Hyperlipoproteinemia Type I
• Hyperlipoproteinemias
• Lipoprotein Lipase Deficiency
• Syndrome

Intervention

Drug:
• AKCEA-ANGPTL3-LRx
AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.

Locations

Country	Name	City	State
Canada	Investigative Site	Montréal	Quebec

Sponsors (2)

Lead Sponsor	Collaborator
Akcea Therapeutics	Ionis Pharmaceuticals, Inc.

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)	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.	Baseline to Month 3
Primary	Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)	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.	Baseline to Month 3
Secondary	Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)	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.	Baseline to Month 3
Secondary	Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)	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.	Baseline to Month 3
Secondary	Fasting Lipid and Lipoprotein Measurements at 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.	Month 3
Secondary	Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters	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.	Baseline to Month 3
Secondary	Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters	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.	Baseline to Month 3
Secondary	Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)	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.	Baseline to Day 92
Secondary	Number of Participants Who Experienced Abdominal Pain During the Treatment Period		Days 1, 29, 57 and 92
Secondary	Number of Participants With Treatment Emergent Adverse Events (TEAEs)	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.	From time of informed consent to end of follow-up period (Up to Week 26)