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

Akcea Therapeutics

Status and phase: Completed; Phase 2

Conditions: Hyperlipoproteinemia Type 1; Lipoprotein Lipase Deficiency; Familial Chylomicronemia Syndrome

Treatments: Drug: AKCEA-ANGPTL3-LRx

Study type: Interventional

Funder types: Industry

Identifiers

NCT03360747

ISIS 703802-CS3

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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3 participants in 1 patient group

AKCEA-ANGPTL3-LRx 20 mg

Experimental group

Description:

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.

Treatment:

Drug: AKCEA-ANGPTL3-LRx