The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
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An Open-Label Study of Volanesorsen (IONIS 304801) Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
Condition or disease
Familial Chylomicronemia SyndromeLipoprotein Lipase DeficiencyHyperlipoproteinemia Type 1
This is a multi-center, open-label study for Group 1: ISIS 304801-CS6 (index study) roll-over FCS patients, Group 2: ISIS 304801-CS16 (index study) roll-over FCS patients, and Group 3: FCS patients who did not participate in the ISIS 304801-CS6 or ISIS 304801-CS16 index studies. All patients will receive volanesorsen 300 mg once per week for 52 weeks. Patients will have the option of continuing dosing for an additional 52 weeks until an expanded access program is approved and available in their country.
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Ages Eligible for Study:
18 Years and older (Adult, Senior)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Must give written informed consent to participate in the study (signed and dated) and any authorization required by law
Able and willing to participate in a 65-week study
Group 1 and 2:
Satisfactory completion of ISIS 304801-CS6 or ISIS 304801-CS16 (index studies) with an acceptable safety profile, per Sponsor and Investigator judgment
Patients who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of Familial Chylomicronemia Syndrome (FCS) may enroll in the study.
History of chylomicronemia
A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
Fasting triglycerides ≥ 750 mg/dL (8.4mmol/L) at Screening
Unwilling to comply with lifestyle requirements for the duration of the study
Group 1 and 2:
Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.
Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
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
Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.