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Completed

NCT Number: NCT02658175

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

An open-label study of volanesorsen (ISIS 304801) administered subcutaneously to participants with FCS.

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Key information

Conditions

Familial Chylomicronemia Syndrome Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Familial hyperchylomicronemia syndrome Genetic Diseases, Inborn Hyperlipidemias Hyperlipoproteinemia Type 1 Hyperlipoproteinemia Type I Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipoprotein Lipase Deficiency Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

IONIS Investigative Site, São Paulo, Brazil

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About this study

This is a multi-center, open-label study for FCS participants rolling over from the ISIS 304801-CS6 (NCT02211209) index study, FCS participants rolling over from the ISIS 304801-CS16 (NCT02300233) index study and Treatment-naïve group. All participants were to receive volanesorsen 300 milligrams (mg) once per week for 52 weeks. Participants were allowed dose adjustment/dose reduction based on monitoring rules. Participants had the option of continuing dosing for an additional 52 weeks (France: up to an additional 104 weeks for a total of 156 weeks) 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 (France: 26-week) post-treatment evaluation period.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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 (NCT02211209) or ISIS 304801-CS16 (NCT02300233) index studies with an acceptable safety profile, per Sponsor and Investigator judgment.

Group 3:

  • Participants who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of FCS may enroll in the study.
  • 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

  • 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 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.

Treatment and study plan

Volanesorsen

Drug

300 mg volanesorsen administered via SC injection.

Other names: IONIS-APOCIIIRx, ISIS 304801

Primary outcomes

  1. Mean Percent Change From Baseline in Fasting Triglyceride (TG)

    Time frame: Baseline and Months 3, 6, and 12

    Baseline for treatment-naïve group was defined as the average of open-label Day 1 pre-dose assessment and the last measurement prior to open-label Day 1. Baseline for CS6-volanesorsen and CS16-volanesorsen arm groups was defined as the average of index study Day 1 pre-dose assessment and the last measurement prior index study Day 1. The values at the Month 3 analysis time point were defined as the average of the Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. The Month 6 analysis time point was at the end of Month 6, and the values were defined as the average of the Week 25 (Day 169) and Week 26 (Day 176) fasting assessments. The values at the Month 12 analysis time point were defined as the average of the Week 50 (Day 344) and Week 52 (Day 358) fasting assessments.

  2. Number of Participants With Treatment-emergent Adverse Events (TEAEs)

    Time frame: From first dose of study drug to end of follow-up period [Up to Week 182]

    An adverse event (AE) was defined as any unfavorable and unintended sign (including a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the study or use of investigational drug product, whether or not the AE was considered related to the investigational drug product. A TEAE was defined as any AE starting or getting worse on or after the first dose of the study drug.

Sponsors and collaborators

Lead sponsor

Akcea Therapeutics

Industry

Collaborators

  • Ionis Pharmaceuticals, Inc.

Registry information

Official study 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)

Important dates

Study start
2015
Primary completion
2020
Study completion
2020
First posted
Jan 18, 2016
Registry last updated
Aug 26, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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