Phase 3
N=67
The APPROACH Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
Familial Chylomicronemia Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02211209 ↗Enrolled (actual)
67
Serious AEs
18.2%
Results posted
Apr 2022
Primary outcome: Primary: Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3 — 17.6; -76.5 percent change — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Volanesorsen (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Ionis Pharmaceuticals, Inc.
- Primary completion
- Dec 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3 |
17.6; -76.5 | < 0.0001 sig |
| SECONDARY Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h) |
36.92; -234.77 | — |
| SECONDARY Absolute Change From Baseline in Fasting TG at Month 3 |
92; -1712 | < 0.0001 sig |
| SECONDARY Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3 |
3; 23 | 0.0001 sig |
| SECONDARY Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3 |
3; 29 | <0.0001 sig |
| SECONDARY Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period |
19; 18; 1; 4; 5; 6 | — |
| SECONDARY Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period |
2.04; 2.73 | 0.6131 |
| SECONDARY Change From Baseline in Hepatosplenomegaly as Assessed by MRI at Week 52 |
-25; 113 | 0.1206 |
Summary
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT02211209). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.