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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02658175
Recruitment Status : Active, not recruiting
First Posted : January 18, 2016
Last Update Posted : January 10, 2020
Sponsor:
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Allocation: N/A
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


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




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 ]


Information from the National Library of Medicine

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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.

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


Locations
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United States, California
IONIS Investigative Site
Huntington Beach, California, United States, 94143
IONIS Investigative Site
San Francisco, California, United States, 94143
United States, Florida
IONIS Investigative Site
Boca Raton, Florida, United States, 33434
United States, Massachusetts
IONIS Investigative Site
Boston, Massachusetts, United States, 02114
United States, Pennsylvania
IONIS Investigative Site
Philadelphia, Pennsylvania, United States, 19104
United States, Texas
IONIS Investigative Site
Houston, Texas, United States, 77030
United States, Virginia
IONIS Investigative Site
Norfolk, Virginia, United States, 23510
United States, Washington
IONIS Investigative Site
Seattle, Washington, United States, 98104
Brazil
IONIS Investigative Site
Sao Paulo, Brazil, 04040-001
IONIS Investigative Site
Sao Paulo, Brazil, CEP-05403-000
Canada, British Columbia
IONIS Investigative Site
Vancouver, British Columbia, Canada, V6Z1Y6
Canada, Quebec
IONIS Investigative Site
Chicoutimi, Quebec, Canada, G7H 7K9
IONIS Investigative Site
Montreal, Quebec, Canada, H2W 1R7
Canada
IONIS Investigative Site
Quebec, Canada, G1V 4W2
France
IONIS Investigative Site
Paris, Cedex 13, France, 75013
IONIS Investigative Site
Marseille Cedex 05, France, 13385
IONIS Investigative Site
Nantes cedex 1, France, 44800
Germany
IONIS Investigative Site
Berlin, Germany, 13353
Israel
IONIS Investigative Site
Safed, Israel, 13110
Italy
IONIS Investigative Site
Palermo, Italy, 90127
IONIS Investigative Site
Roma, Italy, 00161
IONIS Investigative Site
Rome, Italy, 00161
Netherlands
IONIS Investigative Site
Amsterdam-Zuidoost, Netherlands, 1105 AZ
South Africa
IONIS Investigative Site
Cape Town, South Africa, 7925
Spain
IONIS Investigative Site
Barcelona, Spain, 08036
IONIS Investigative Site
La Coruna, Spain, 15001
IONIS Investigative Site
Madrid, Spain, 28007
IONIS Investigative Site
Sevilla, Spain, 41013
IONIS Investigative Site
Zaragoza, Spain, 50009
United Kingdom
IONIS Investigative Site
Birmingham, United Kingdom, B9 5SS
IONIS Investigative Site
London, United Kingdom, SE1 7EH
IONIS Investigative Site
Manchester, United Kingdom, M13 9WL
IONIS Investigative Site
Manchester, United Kingdom, M23 9LT
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
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Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02658175    
Other Study ID Numbers: ISIS 304801-CS7
First Posted: January 18, 2016    Key Record Dates
Last Update Posted: January 10, 2020
Last Verified: January 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type I
Syndrome
Hyperlipoproteinemias
Hyperlipidemias
Disease
Pathologic Processes
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn