The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

Ionis Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Familial Chylomicronemia Syndrome

Treatments: Drug: Volanesorsen; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02211209

2014-002421-35 (EudraCT Number)

ISIS 304801-CS6

Details and patient eligibility

About

The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome

Enrollment: 67 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• History of chylomicronemia
• A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
• Fasting triglycerides (TG) ≥ 750 mg/dL (8.4 mmol/L) at Screening

Exclusion criteria

• Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
• Other types of severe hypertriglyceridemia
• Active pancreatitis within 4 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 IONIS-APOCIIIRx
• Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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67 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

Volanesorsen-matching placebo administered subcutaneously once-weekly for 52 weeks.

Treatment:

Drug: Placebo

Volanesorsen

Experimental group

Description:

Volanesorsen 300 mg administered subcutaneously once-weekly for 52 weeks.

Treatment:

Drug: Volanesorsen