The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
NCT ID: NCT02658175
Last Updated: 2021-08-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
68 participants
INTERVENTIONAL
2015-12-23
2020-01-15
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Treatment-naïve Group
Treatment naïve group included combined group of ISIS 304801-CS7 (CS7-New) study participant and participant on placebo in index studies (ISIS 304801-CS6 \[NCT02211209\] and ISIS 304801-CS16 \[NCT02300233\]), were to receive 300 mg of volanesorsen as single SC once weekly for Weeks 1-52 of this study. Participants were allowed dose adjustment/dose reduction based on monitoring rules. Following Week 52 visit, participants had option of participating in expanded access program or continuing treatment with 300 mg of volanesorsen as single SC once-weekly for up to additional 52 weeks (Weeks 53-104) and in France participants, up to additional 104 weeks for total of 156 weeks (Weeks 105 to Week 156) until expanded access program was approved and available in their country. Participants who were not participating in expanded access program were to enter 13-week post-treatment (PT) evaluation period and in France, participants not continuing treatment were to enter 26-week PT follow-up period.
Volanesorsen
300 mg volanesorsen administered via SC injection.
CS6-Volanesorsen
Participants with FCS rolling over from the ISIS 304801-CS6 (NCT02211209) index study after receiving volanesorsen, were to receive 300 mg of volanesorsen as a single SC injection once weekly for Weeks 1-52 of this study. Participants were allowed dose adjustment/dose reduction based on monitoring rules. Following the Week 52 visit, participants had the option of participating in an expanded access program or continuing treatment with 300 mg of volanesorsen as a single SC injection once-weekly for up to an additional 52 weeks (Weeks 53-104) and in France participants, up to an additional 104 weeks for total of 156 weeks of treatment (Weeks 105 to Week 156) of this study until an expanded access program was approved and available in their country. Participants who were not participating in an expanded access program were to enter a 13-week post-treatment evaluation period and in France, participants not continuing treatment were to enter a 26-week post-treatment follow-up period.
Volanesorsen
300 mg volanesorsen administered via SC injection.
CS16-Volanesorsen
Participants with FCS rolling over from the ISIS 304801-CS16 (NCT02300233) index study after receiving volanesorsen, were to receive 300 mg of volanesorsen as a single SC injection once weekly for Weeks 1-52 of this study. Participants were allowed dose adjustment/dose reduction based on monitoring rules. Following the Week 52 visit, participants had the option of participating in an expanded access program or continuing treatment with 300 mg of volanesorsen as a single SC injection once-weekly for up to an additional 52 weeks (Weeks 53-104) and in France participants, up to an additional 104 weeks for total of 156 weeks of treatment (Weeks 105 to Week 156) of this study until an expanded access program was approved and available in their country. Participants who were not participating in an expanded access program were to enter a 13-week post-treatment evaluation period and in France, participants not continuing treatment were to enter a 26-week post-treatment follow-up period.
Volanesorsen
300 mg volanesorsen administered via SC injection.
Interventions
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Volanesorsen
300 mg volanesorsen administered via SC injection.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Able and willing to participate in a 65-week study.
Group 1 and 2:
* Satisfactory completion of ISIS 304801-CS6 (NCT02211209) or ISIS 304801-CS16 (NCT02300233) index studies with an acceptable safety profile, per Sponsor and Investigator judgment.
Group 3:
* History of chylomicronemia.
* A diagnosis of FCS (Type 1 Hyperlipoproteinemia.)
* Fasting triglycerides greater than or equal to (≥)750 milligrams per deciliter \[mg/dL\] (8.4 millimoles per liter \[mmol/L\]) at Screening.
Exclusion Criteria
Group 1 and 2:
* Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study.
Group 3:
* Diabetes mellitus if newly diagnosed or if hemoglobin A1c (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 participant participating in or completing the study.
18 Years
ALL
No
Sponsors
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Ionis Pharmaceuticals, Inc.
INDUSTRY
Akcea Therapeutics
INDUSTRY
Responsible Party
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Locations
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IONIS Investigative Site
Huntington Beach, California, United States
IONIS Investigative Site
San Francisco, California, United States
IONIS Investigative Site
Boca Raton, Florida, United States
IONIS Investigative Site
Boston, Massachusetts, United States
IONIS Investigative Site
Philadelphia, Pennsylvania, United States
IONIS Investigative Site
Houston, Texas, United States
IONIS Investigative Site
Norfolk, Virginia, United States
IONIS Investigative Site
Seattle, Washington, United States
IONIS Investigative Site
São Paulo, , Brazil
IONIS Investigative Site
São Paulo, , Brazil
IONIS Investigative Site
Vancouver, British Columbia, Canada
IONIS Investigative Site
Chicoutimi, Quebec, Canada
IONIS Investigative Site
Montreal, Quebec, Canada
IONIS Investigative Site
Québec, , Canada
IONIS Investigative Site
Paris, Cedex 13, France
IONIS Investigative Site
Marseille, , France
IONIS Investigative Site
Nantes, , France
IONIS Investigative Site
Berlin, , Germany
IONIS Investigative Site
Cologne, , Germany
IONIS Investigative Site
Safed, , Israel
IONIS Investigative Site
Palermo, , Italy
IONIS Investigative Site
Roma, , Italy
IONIS Investigative Site
Rome, , Italy
IONIS Investigative Site
Amsterdam-Zuidoost, , Netherlands
IONIS Investigative Site
Cape Town, , South Africa
IONIS Investigative Site
A Coruña, , Spain
IONIS Investigative Site
Barcelona, , Spain
IONIS Investigative Site
Madrid, , Spain
IONIS Investigative Site
Seville, , Spain
IONIS Investigative Site
Zaragoza, , Spain
IONIS Investigative Site
Birmingham, , United Kingdom
IONIS Investigative Site
London, , United Kingdom
IONIS Investigative Site
Manchester, , United Kingdom
IONIS Investigative Site
Manchester, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2015-003755-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
ISIS 304801-CS7
Identifier Type: -
Identifier Source: org_study_id
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