The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in participants with Familial Chylomicronemia Syndrome
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
67
Percent Change in Fasting Triglycerides (TG) From Baseline to Month 3
The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.
Time frame: Baseline to 3 months
Change From Baseline in Postprandial TG Area Under the Curve (AUC)(0-9h)
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.
Time frame: Baseline to an on-treatment assessment between Week 13 and Week 19
Absolute Change From Baseline in Fasting TG at Month 3
The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.
Time frame: Baseline to 3 months
Treatment Response Rate Defined as Participants With Fasting Plasma TG < 750 mg/dL at Month 3
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
Time frame: Baseline to 3 months
Treatment Response Rate Defined as Participants With Fasting TG ≥ 40% Reduction From Baseline at Month 3
The Month 3 endpoint was defined as the average of Week 12 (Day 78) and Week 13 (Day 85) fasting assessments.
Time frame: Baseline to 3 months
Frequency and Severity of Participant-reported Abdominal Pain During the Treatment Period
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
IONIS Investigative Site
Encinitas, California, United States
IONIS Investigative Site
San Francisco, California, United States
IONIS Investigative Site
Kansas City, Kansas, United States
IONIS Investigative Site
Boston, Massachusetts, United States
IONIS Investigative Site
New York, New York, United States
IONIS Investigative Site
Oklahoma City, Oklahoma, United States
IONIS Investigative Site
Portland, Oregon, United States
IONIS Investigative Site
Philadelphia, Pennsylvania, United States
IONIS Investigative Site
Houston, Texas, United States
IONIS Investigative Site
Norfolk, Virginia, United States
...and 30 more locations
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.
Time frame: Baseline to 12 months
Frequency of the Composite of Episodes of Acute Pancreatitis and Participant-reported Moderate/Severe Abdominal Pain During the Treatment Period
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.
Time frame: 12 months
Change From Baseline in Hepatosplenomegaly as Assessed by MRI at 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.
Time frame: Baseline to Week 52