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Completed

NCT Number: NCT03360747

Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)

This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in 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 2

Primary location

Investigative Site

Montreal, Quebec, H2W 1R7, Canada

Who can participate

Healthy volunteers accepted: No

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

Key Inclusion Criteria:

  • Genetically confirmed chylomicronemia syndrome.
  • Fasting triglycerides greater than or equal to (>=) 750 milligrams per deciliter (mg/dL) [8.4 millimoles per liter (mmol/L)] at Screening.

Key Exclusion Criteria:

  • Diabetes mellitus if newly diagnosed or if glycated hemoglobin (HbA1c) >= 9.0%.
  • Active pancreatitis within 2 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 AKCEA-ANGPTL3-LRx.
  • Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.

Treatment and study plan

AKCEA-ANGPTL3-LRx

Drug

AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.

Other names: ISIS 703802, IONIS-ANGPTL3-LRx, Vupanorsen

Primary outcomes

  1. Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)

    Time frame: Baseline to Month 3

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  2. Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)

    Time frame: Baseline to Month 3

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

Secondary outcomes

  1. Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)

    Time frame: Baseline to Month 3

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  2. Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)

    Time frame: Baseline to Month 3

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  3. Fasting Lipid and Lipoprotein Measurements at Month 3

    Time frame: Month 3

    Fasting lipid and lipoprotein measurements included non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C and LDL-C. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  4. Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters

    Time frame: Baseline to Month 3

    Other fasting lipid measurements included total cholesterol (TC), non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  5. Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters

    Time frame: Baseline to Month 3

    Other fasting lipid measurements included TC, non-HDL-C, ApoB, HDL-C, ApoA-1, VLDL-C, and LDL-C. Baseline was defined as average of Day 1 predose fasting assessment and last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

  6. Change From Baseline to Day 92 in Maximum Postprandial Triglycerides (TG)

    Time frame: Baseline to Day 92

    Participants consumed standardized pre-cooked meals (lunches and dinners and instructions for breakfasts and snacks) for 2 days prior to the postprandial assessments. Change from Baseline to Day 92 in maximum postprandial TG was assessed.

  7. Number of Participants Who Experienced Abdominal Pain During the Treatment Period

    Time frame: Days 1, 29, 57 and 92

  8. Number of Participants With Treatment Emergent Adverse Events (TEAEs)

    Time frame: From time of informed consent to end of follow-up period (Up to Week 26)

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

A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRx (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Important dates

Study start
2017
Primary completion
2018
Study completion
2018
First posted
Dec 4, 2017
Registry last updated
Jan 7, 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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