Familial Chylomicronemia SyndromePhase 2NCT03360747

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

A Phase 2 study testing AKCEA-ANGPTL3-LRx in people with Familial Chylomicronemia Syndrome and Lipoprotein Lipase Deficiency, run by Akcea Therapeutics at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
What is being tested?: AKCEA-ANGPTL3-LRx
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 3 participants
Where: 1 siteCanada
Study dates: 21 Dec 2017to 4 Sep 2018
Phase: Phase 2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

1 site in 1 country.

Canada1 site

Investigative Site
Montreal, Quebec, H2W 1R7

Other trials in Canada

About this study

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)

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.

Conditions: Familial Chylomicronemia Syndrome, Lipoprotein Lipase Deficiency, Hyperlipoproteinemia Type 1
Being tested: AKCEA-ANGPTL3-LRx
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
Medical subject headings: Familial hyperchylomicronemia syndrome, Hyperlipoproteinemia Type I, Hyperlipoproteinemias, Hyperlipidemias, Dyslipidemias, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Metabolic Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AKCEA-ANGPTL3-LRxDrug
Also called ISIS 703802, IONIS-ANGPTL3-LRx, Vupanorsen
AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.

Study groups in NCT03360747
Study group	Role	What they receive
AKCEA-ANGPTL3-LRx 20 mg	Experimental	Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26. Receives: Drug: AKCEA-ANGPTL3-LRx

What is being measured

The primary outcome is the main question the study is designed to answer.

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.
Measured: Baseline to Month 3
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.
Measured: Baseline to Month 3

Secondary outcomes (8)

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.
Measured: Baseline to Month 3
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.
Measured: Baseline to Month 3
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.
Measured: Month 3
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.
Measured: Baseline to Month 3
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.
Measured: Baseline to Month 3
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.
Measured: Baseline to Day 92
Number of Participants Who Experienced Abdominal Pain During the Treatment Period
Measured: Days 1, 29, 57 and 92
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.
Measured: From time of informed consent to end of follow-up period (Up to Week 26)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

21 Dec 20174 Sep 2018

First posted4 Dec 2017
Study start21 Dec 2017
Primary completion12 Jun 2018
Study completion4 Sep 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Familial Chylomicronemia SyndromeStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03360747
Sponsor's protocol ID: ISIS 703802-CS3
Status last verified: Dec 2020
First submitted: 18 Nov 2017
Last updated: 7 Jan 2021
Results posted: Yes, on ClinicalTrials.gov
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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