Trial · NCT04568434
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
NCT04568434, Phase 3, Completed, Ionis Pharmaceuticals, Inc.; results first posted 2025-03-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS). Trials, record NCT04568434. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04568434
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
- NCT number
- NCT04568434
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 66 participants, actual
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Conditions
- Familial Chylomicronemia Syndrome
- Interventions
- Olezarsen, Placebo
- First posted
- 2020-09-29
- Last updated
- 2025-03-06
- Other registries' IDs
- eudract:2020-002536-67
- Results first posted
- 2025-03-06
Source ClinicalTrials.gov
Registered outcomes15
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percent Change From Baseline in Fasting TG at Month 6 | Baseline, Month 6 | |
| Secondary | Percent Change From Baseline in Fasting TG at Month 12 | Baseline, Month 12 | |
| Secondary | Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Baseline, Months 6 and 12 | |
| Secondary | Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6 | Month 6 | Percentages are rounded off to the nearest single decimal place. |
| Secondary | Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12 | Baseline, Months 6 and 12 | |
| Secondary | Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12 | Baseline, Months 6 and 12 | |
| Secondary | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis | During the treatment period Week 1 through Week 53 | All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the Acute Pancreatitis Adjudication Committee (PAC) Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the treatment period. |
| Secondary | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) | During the treatment period Week 1 through Week 53 | All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the treatment period. |
| Secondary | Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis | Week 13 through Week 53 | All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the specified duration. |
| Secondary | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53 | Week 13 through Week 53 | All AEs and SAEs that consistently occurred during the study with an event of acute pancreatitis were adjudicated by a blinded, independent committee according to the Atlanta classification of acute pancreatitis as outlined in the PAC Charter. These events were categorized as 1) documented pancreatitis, 2) probable pancreatitis, 3) possible pancreatitis, 4) unable to adjudicate and 5) no diagnosis of acute pancreatitis. The adjudicated event rate represents the average number of events per 100 participant-years during the specified duration. |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 registered outcomesOutcomes15
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percent Change From Baseline in Fasting TG at Month 6 | Baseline, Month 6 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Fasting TG at Month 12 | Baseline, Month 12 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Baseline, Months 6 and 12 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With ≥ 40% Reduction in Fasting TG at Month 6 | Month 6 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percentage of participants | NUMBER |
| SECONDARY | Percent Change From Baseline in Fasting Apolipoprotein B-48 (apoB-48) at Months 6 and 12 | Baseline, Months 6 and 12 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Percent Change From Baseline in Fasting Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) at Months 6 and 12 | Baseline, Months 6 and 12 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) in Participants With Prior History of Pancreatitis | During the treatment period Week 1 through Week 53 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. Overall number of participants analyzed is the number of participants with a history of pancreatitis within 10 years prior to screening. As prespecified in the protocol, data for this outcome measure was collected and reported in a pooled manner for olezarsen (combined Olezarsen 50 mg + Olezarsen 80 mg) | events per 100 participant-years | MEAN |
| SECONDARY | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During the Treatment Period (Week 1 Through Week 53) | During the treatment period Week 1 through Week 53 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. As prespecified in the protocol, data for this outcome measure was collected and reported in a pooled manner for olezarsen (combined Olezarsen 50 mg + Olezarsen 80 mg). | events per 100 participant-years | MEAN |
| SECONDARY | Adjudicated Acute Pancreatitis Mean Event Per 100 Participant-Years Rate During Week 13 Through Week 53 in Participants With Prior History of Pancreatitis | Week 13 through Week 53 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. Overall number of participants analyzed is the number of participants with a history of pancreatitis within 10 years prior to screening. As prespecified in the protocol, data for this outcome measure was collected and reported in a pooled manner for olezarsen (combined Olezarsen 50 mg + Olezarsen 80 mg). | events per 100 participant-years | MEAN |
| SECONDARY | Adjudicated Acute Pancreatitis Mean Event Rate Per 100 Participant-Years During Week 13 to Week 53 | Week 13 through Week 53 | FAS included all participants that were randomly assigned to treatment and received at least 1 dose of olezarsen or placebo. As prespecified in the protocol, data for this outcome measure was collected and reported in a pooled manner for olezarsen (combined Olezarsen 50 mg + Olezarsen 80 mg). | events per 100 participant-years | MEAN |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 outcomesResults by group48
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percent Change From Baseline in Fasting TG at Month 6 | Placebo | 11.52 | 95% Confidence Interval -5.321 to 28.356 | percent change | |
| Percent Change From Baseline in Fasting TG at Month 6 | Olezarsen 50 mg | -10.85 | 95% Confidence Interval -27.797 to 6.095 | percent change | |
| Percent Change From Baseline in Fasting TG at Month 6 | Olezarsen 80 mg | -31.99 | 95% Confidence Interval -49.031 to -14.940 | percent change | |
| Percent Change From Baseline in Fasting TG at Month 12 | Placebo | 20.89 | 95% Confidence Interval 1.016 to 40.764 | percent change | |
| Percent Change From Baseline in Fasting TG at Month 12 | Olezarsen 50 mg | -22.92 | 95% Confidence Interval -42.505 to -3.336 | percent change | |
| Percent Change From Baseline in Fasting TG at Month 12 | Olezarsen 80 mg | -38.50 | 95% Confidence Interval -58.187 to -18.815 | percent change | |
| Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Placebo | Month 6 | 7.57 | 95% Confidence Interval -5.229 to 20.359 | percent change |
| Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Olezarsen 50 mg | Month 6 | -57.91 | 95% Confidence Interval -71.193 to -44.631 | percent change |
| Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Olezarsen 80 mg | Month 6 | -66.13 | 95% Confidence Interval -79.437 to -52.823 | percent change |
| Percent Change From Baseline in Fasting Apolipoprotein C-III (apoC-III) at Months 6 and 12 | Placebo | Month 12 | 17.08 | 95% Confidence Interval 2.149 to 32.009 | percent change |
1 to 10 of 48Source ClinicalTrials.gov
See all 48 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Placebo | 23 | |
| Overall Study | STARTED | Olezarsen 50 mg | 21 | |
| Overall Study | STARTED | Olezarsen 80 mg | 22 | |
| Overall Study | COMPLETED | Placebo | 22 | |
| Overall Study | COMPLETED | Olezarsen 50 mg | 19 | |
| Overall Study | COMPLETED | Olezarsen 80 mg | 19 | |
| Overall Study | NOT COMPLETED | Placebo | 1 | |
| Overall Study | NOT COMPLETED | Olezarsen 50 mg | 2 | |
| Overall Study | NOT COMPLETED | Olezarsen 80 mg | 3 |
Source ClinicalTrials.gov
Adverse events141
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anaemia | Blood and lymphatic system disorders | Placebo | 1 | 23 | |
| other | Anaemia | Blood and lymphatic system disorders | Olezarsen 50 mg | 0 | 21 | |
| other | Anaemia | Blood and lymphatic system disorders | Olezarsen 80 mg | 2 | 22 | |
| other | Abdominal distension | Gastrointestinal disorders | Placebo | 0 | 23 | |
| other | Abdominal distension | Gastrointestinal disorders | Olezarsen 50 mg | 0 | 21 | |
| other | Abdominal distension | Gastrointestinal disorders | Olezarsen 80 mg | 2 | 22 | |
| other | Abdominal pain | Gastrointestinal disorders | Placebo | 8 | 23 | |
| other | Abdominal pain | Gastrointestinal disorders | Olezarsen 50 mg | 3 | 21 | |
| other | Abdominal pain | Gastrointestinal disorders | Olezarsen 80 mg | 4 | 22 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 1 | 23 |
1 to 10 of 141Source ClinicalTrials.gov
See all 141 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo | 0 | 23 |
| Total, all-cause mortality | Olezarsen 50 mg | 1 | 21 |
| Total, all-cause mortality | Olezarsen 80 mg | 0 | 22 |
| Total, other adverse events | Placebo | 19 | 23 |
| Total, other adverse events | Olezarsen 50 mg | 17 | 21 |
| Total, other adverse events | Olezarsen 80 mg | 18 | 22 |
| Total, serious adverse events | Placebo | 9 | 23 |
| Total, serious adverse events | Olezarsen 50 mg | 4 | 21 |
| Total, serious adverse events | Olezarsen 80 mg | 3 | 22 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04568434
GET https://conductscience.org/api/trials/v1/NCT04568434/results?page=2