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

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ClinicalTrials.gov Identifier: NCT02658175
Recruitment Status : Active, not recruiting
First Posted : January 18, 2016
Last Update Posted : November 16, 2018
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Familial Chylomicronemia Syndrome Lipoprotein Lipase Deficiency Hyperlipoproteinemia Type 1 Drug: Volanesorsen Phase 3

Detailed Description:
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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : December 31, 2015
Estimated Primary Completion Date : June 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Volanesorsen Drug: Volanesorsen
300 mg volanesorsen
Other Names:
  • IONIS-APOCIIIRx
  • ISIS 304801


Outcome Measures
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Primary Outcome Measures :
  1. Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 117 Weeks ]

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

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02658175


  Show 33 Study Locations
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
More Information
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02658175     History of Changes
Other Study ID Numbers: ISIS 304801-CS7
First Posted: January 18, 2016    Key Record Dates
Last Update Posted: November 16, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes

Additional relevant MeSH terms:
Syndrome
Hyperlipoproteinemias
Hyperlipidemias
Hyperlipoproteinemia Type I
Disease
Pathologic Processes
 Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn