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Summary

for people ages 18 years and up (full criteria)
at Huntington Beach, California and other locations
study started
estimated completion:

Description

Summary

An Open-Label Study of Volanesorsen (IONIS 304801) Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)

Official Title

ISIS 304801-CS7 The APPROACH Open Label Study Volanesorsen (ISIS 304801) An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Details

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.

Keywords

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

Eligibility

You can join if…

Open to people ages 18 years and up

  • 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

Group 3:

  • 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

You CAN'T join if...

  • 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.

Group 3:

  • 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.

Locations

  • IONIS Investigative Site accepting new patients
    Huntington Beach, California, 94143, United States
  • IONIS Investigative Site not yet accepting patients
    San Francisco, California, 94143, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Ionis Pharmaceuticals, Inc.
ID
NCT02658175
Phase
Phase 3
Lead Scientist
Eveline Stock
Study Type
Interventional
Last Updated
July 26, 2017
I’m interested in this study!