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Completed

NCT Number: NCT02211209

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

The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome

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Key information

Conditions

Familial Chylomicronemia Syndrome Familial hyperchylomicronemia syndrome

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

IONIS Investigative Site, Campinas, Brazil

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • History of chylomicronemia
  • A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
  • Fasting triglycerides (TG) ≥ 750 mg/dL (8.4 mmol/L) at Screening

Exclusion criteria

  • Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
  • Other types of severe hypertriglyceridemia
  • 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
  • Previous treatment with IONIS-APOCIIIRx
  • 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

Other names: ISIS 304801, ApoC-III, Approach, IONIS-APOCIIIRx

Placebo

Drug

Primary outcomes

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

    Time frame: Baseline to 3 months

    The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.

Secondary outcomes

  1. Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h)

    Time frame: Baseline to an on-treatment assessment between Week 13 and Week 19

    Participants had 2 postprandial assessments - one at Baseline (completed at least 48 hours prior to first dose) and one at any time between Week 13 and 19, inclusive. Assessment timepoints include from 1-hr before to up to 9 hrs after ingestion of the meal at 1-hour interval. Postprandial AUC results were calculated using a linear trapezoidal rule for each postprandial measure in the subset of participants who had postprandial assessments 0-9 hour results at baseline and the postbaseline between Week 13 to 19.

  2. Absolute Change From Baseline in Fasting TG at Month 3

    Time frame: Baseline to 3 months

    The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.

  3. Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3

    Time frame: Baseline to 3 months

    The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments. mg/dL = milligrams per deciliter

  4. Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3

    Time frame: Baseline to 3 months

    The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.

  5. Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period

    Time frame: Baseline to 12 months

    Abdominal pain was measured according to the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25. Missing data were imputed by using next observation carried back (NOCB) if there was a subsequent score available.

  6. Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period

    Time frame: 12 months

    Moderate/severe abdominal pain was defined as having a pain score of 4-10 on the Bracket electronic patient-reported outcomes (ePRO) assessment. Scores were categorized as follows: no pain (pain score: 0), mild (pain score: 1-3), moderate (pain score: 4-6), or severe (pain score: 7-10). The yearly frequency was calculated as the number of episodes during the on-treatment period / (last dose date - first dose date + 28) * 365.25.

  7. Change From Baseline in Hepatosplenomegaly as Assessed by MRI at Week 52

    Time frame: Baseline to Week 52

    The Week 52 endpoint was defined as the average of Week 50 (Day 344)/Week 51 (Day 351) and Week 52 (Day 358) fasting assessments.

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Collaborators

  • Akcea Therapeutics

Registry information

Official study title

A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Important dates

Study start
2014
Primary completion
2016
Study completion
2017
First posted
Aug 7, 2014
Registry last updated
Apr 13, 2022

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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