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

This study has been completed.
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc. Identifier:
First received: August 5, 2014
Last updated: May 1, 2017
Last verified: May 2017
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in patients with Familial Chylomicronemia Syndrome

Condition Intervention Phase
Familial Chylomicronemia Syndrome Drug: volanesorsen Drug: Placebo Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Resource links provided by NLM:

Further study details as provided by Ionis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Efficacy of ISIS-APOCIIIRx as measured by the percent change in fasting triglycerides from baseline [ Time Frame: 13 Weeks ]

Enrollment: 67
Study Start Date: August 2014
Study Completion Date: March 27, 2017
Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: volanesorsen
300 mg volanesorsen administered subcutaneously once-weekly for 52 weeks
Drug: volanesorsen
Other Names:
  • ISIS 304801
  • ApoC-III
  • Approach
Placebo Comparator: Placebo
Placebo administered subcutaneously once-weekly for 52 weeks
Drug: Placebo


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • History of chylomicronemia
  • A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
  • Fasting triglycerides ≥ 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 patient participating in or completing the study
  Contacts and Locations
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Please refer to this study by its identifier: NCT02211209

  Show 42 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
  More Information

Responsible Party: Ionis Pharmaceuticals, Inc. Identifier: NCT02211209     History of Changes
Other Study ID Numbers: ISIS 304801-CS6
Study First Received: August 5, 2014
Last Updated: May 1, 2017

Additional relevant MeSH terms:
Hyperlipoproteinemia Type I
Pathologic Processes
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders
Metabolic Diseases processed this record on July 27, 2017