Trial · NCT02527343
The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
NCT02527343, Phase 2, Phase 3, Terminated, Akcea Therapeutics; results first posted 2021-10-18
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy. Trials, record NCT02527343. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02527343
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
- NCT number
- NCT02527343
- Status
- Terminated
- Phases
- Phase 2, Phase 3
- Enrollment
- 40 participants, actual
- Lead sponsor
- Akcea Therapeutics
- Conditions
- Familial Partial Lipodystrophy
- Interventions
- volanesorsen, Placebo
- First posted
- 2015-08-19
- Last updated
- 2021-10-18
- Other registries' IDs
- eudract:2015-000493-35
- Results first posted
- 2021-10-18
- Limitations and caveats
- The Sponsor decided to terminate the study early at a time point when sufficient data had been accumulated to inform a decision on further development of volanesorsen in participants with FPL.
Source ClinicalTrials.gov
Registered outcomes16
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | 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 predose fasting assessment. Month 3 value was defined as the average of Week 12 and Week 13 fasting TG assessments of the randomized treatment period. The data was analyzed using an analysis of covariance (ANCOVA) model with the randomization stratification factor (diagnosis of disease with or without genetics and family history) and treatment group as factors and log-transformed baseline fasting TG as a covariate. |
| Secondary | Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Baseline, Months 6 and 12 | Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Randomized treatment period: Month 6 value was defined as Week 25 or Week 26 for MRI assessment and Month 12 was defined as Week 50 or Week 52 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI. |
| Secondary | Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Baseline, Months 6 and 12 | Baseline was defined as the last non-missing assessment prior to the first dose of study drug in the randomized treatment period. Open-label extension period: Month 6 value was defined as Week 77 or Week 78 for MRI assessment and Month 12 value was defined as Week 102 or Week 104 for MRI assessment. Hepatic steatosis is a reversible condition in which large vacuoles of triglyceride fat accumulate in the liver cells, causing nonspecific inflammation. Hepatic Steatosis was assessed by hepatic fat fraction using MRI. |
| Secondary | Randomized Treatment Period: Change From Baseline in Hemoglobin A1c (HbA1c) | Baseline, Months 3, 6, 9, and 12 | Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: The Month 3 value was defined as Week 13, Month 6 value was defined as Week 26, Month 9 value was defined as Week 38 and Month 12 value was defined as Week 52. |
| Secondary | Open Label Extension Period: Change From Baseline in HbA1c | Baseline, Months 3, 6, 9, and 12 | Baseline was defined as the last non-missing assessment prior to the first dose of study drug. Open-label extension period: Month 3 value was defined as Week 65, Month 6 value was defined as Week 78, Month 9 value was defined as Week 90 and Month 12 value was defined as Week 104. |
| Secondary | Randomized Treatment Period: Percentage of Participants Who Achieved Greater Than or Equal to (≥) 40% Reduction in Fasting Triglyceride and ≥ 30% Reduction of Hepatic Fat Fraction at Month 6 | Month 6 | The baseline of TG is defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. The baseline of hepatic fat fraction is defined as the last non-missing assessment prior to the first dose of study drug. Randomized treatment period: Month 6 value was defined as average of Week 25 and Week 26 for fasting TG and Week 25 or Week 26 for hepatic fat fraction. |
| Secondary | Randomized Treatment Period: Change From Baseline in Disease Burden Score | From the first dose of study drug to Week 52 | The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities. |
| Secondary | Open-Label Extension Period: Change From Baseline in Disease Burden Score | From the first dose of study drug in open label extension period to Week 117 | The Disease Burden Score is a questionnaire that allows participants to self-report their chronic conditions and then assess the degree to which each condition interferes with daily activities. |
| Secondary | Randomized Treatment Period: Patient-Reported Pain | From the first dose of study drug up to Week 52 | Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories. |
| Secondary | Open Label Extension Period: Patient-Reported Pain | From the first dose of study drug in open label extension period up to Week 117 | Patient-reported pain was assessed by rating pain symptoms at its worst and least for the last 24 hours, on average, and at the moment, with 0 as the lowest score (no pain) and 10 as the highest score (worst pain as you can imagine). Patient-reported pain was also assessed by rating pain symptoms (rate pain on average, rate pain right now) that interfered with general activity, interfered with mood, walking ability, normal work, relations with other people, sleep, and enjoyment of life, with 0 as the lowest score (did not interfered) and 10 as the highest score (completely interfered). The scores from each assessment time point were averaged for all of the below reported categories. |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 registered outcomesOutcomes16
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Baseline to Month 3 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. This outcome measure is reported here for the randomized treatment period only, as per the planned analysis. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Baseline, Months 6 and 12 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. | percent change | LEAST_SQUARES_MEAN |
| SECONDARY | Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Baseline, Months 6 and 12 | FAS included all participants who were randomized and received at least one dose of study drug in the open-label extension period, and who had a baseline fasting TG assessment. Here, number analyzed signifies participants evaluable for this outcome measure at specified time points. | percent change | MEAN |
| SECONDARY | Randomized Treatment Period: Change From Baseline in Hemoglobin A1c (HbA1c) | Baseline, Months 3, 6, 9, and 12 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. | percentage of HbA1c | LEAST_SQUARES_MEAN |
| SECONDARY | Open Label Extension Period: Change From Baseline in HbA1c | Baseline, Months 3, 6, 9, and 12 | FAS included all participants who were randomized and received at least one dose of study drug in the open-label extension period, and who had a baseline fasting TG assessment. Here, number analyzed signifies participants evaluable for this outcome measure at specified time points. | percentage of HbA1c | MEAN |
| SECONDARY | Randomized Treatment Period: Percentage of Participants Who Achieved Greater Than or Equal to (≥) 40% Reduction in Fasting Triglyceride and ≥ 30% Reduction of Hepatic Fat Fraction at Month 6 | Month 6 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. This outcome measure is reported here for the randomized treatment period only, as per the planned analysis. | percentage of participants | NUMBER |
| SECONDARY | Randomized Treatment Period: Change From Baseline in Disease Burden Score | From the first dose of study drug to Week 52 | Data for this outcome measure was not collected due to the change in planned analysis. | ||
| SECONDARY | Open-Label Extension Period: Change From Baseline in Disease Burden Score | From the first dose of study drug in open label extension period to Week 117 | Data for this outcome measure was not collected due to the change in planned analysis. | ||
| SECONDARY | Randomized Treatment Period: Patient-Reported Pain | From the first dose of study drug up to Week 52 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. | score on a scale | MEAN |
| SECONDARY | Open Label Extension Period: Patient-Reported Pain | From the first dose of study drug in open label extension period up to Week 117 | FAS included all participants who were randomized and received at least one dose of study drug in the randomized treatment period, and who had a baseline fasting TG assessment. Here, overall number of participants analyzed signifies participants who were evaluable for this outcome measure. | score on a scale | MEAN |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 outcomesResults by group242
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Randomized Treatment Period: Placebo | -21.64 | 95% Confidence Interval -60.85 to 17.57 | percent change | |
| Randomized Treatment Period: Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG) | Randomized Treatment Period: Volanesorsen | -88.47 | 95% Confidence Interval -133.56 to -43.38 | percent change | |
| Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Randomized Treatment Period: Placebo | Percent Change at Month 6 | 2.83 | 95% Confidence Interval -23.46 to 29.12 | percent change |
| Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Randomized Treatment Period: Volanesorsen | Percent Change at Month 6 | -22.86 | 95% Confidence Interval -52.07 to 6.34 | percent change |
| Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Randomized Treatment Period: Placebo | Percent Change at Month 12 | 1.46 | 95% Confidence Interval -30.49 to 33.42 | percent change |
| Randomized Treatment Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using Magnetic Resonance Imaging (MRI) | Randomized Treatment Period: Volanesorsen | Percent Change at Month 12 | -51.87 | 95% Confidence Interval -87.87 to -15.87 | percent change |
| Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Open-Label Extension Period: Placebo/Volanesorsen | Percent Change at Month 6 | -18.4 | Standard Deviation 54.7 | percent change |
| Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Open-Label Extension Period: Volanesorsen/Volanesorsen | Percent Change at Month 6 | -60.2 | Standard Deviation 43.6 | percent change |
| Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Open-Label Extension Period: Placebo/Volanesorsen | Percent Change at Month 12 | -93.5 | Standard Deviation 44.4 | percent change |
| Open-Label Extension Period: Percent Change From Baseline in Hepatic Steatosis as Assessed by Hepatic Fat Fraction Using MRI | Open-Label Extension Period: Volanesorsen/Volanesorsen | Percent Change at Month 12 | -22.1 | Standard Deviation 47.5 | percent change |
1 to 10 of 242Source ClinicalTrials.gov
See all 242 results by groupParticipant flow90
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| RT Period: Weeks 1 to 52 | STARTED | Randomized Treatment Period: Placebo | 19 | |
| RT Period: Weeks 1 to 52 | STARTED | Randomized Treatment Period: Volanesorsen | 21 | |
| RT Period: Weeks 1 to 52 | STARTED | Randomized Post-Treatment Follow-up Period: Placebo | 0 | |
| RT Period: Weeks 1 to 52 | STARTED | Randomized Post-Treatment Follow-up Period: Volanesorsen | 0 | |
| RT Period: Weeks 1 to 52 | STARTED | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | 0 | |
| RT Period: Weeks 1 to 52 | STARTED | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen | 0 | |
| RT Period: Weeks 1 to 52 | COMPLETED | Randomized Treatment Period: Placebo | 13 | |
| RT Period: Weeks 1 to 52 | COMPLETED | Randomized Treatment Period: Volanesorsen | 14 | |
| RT Period: Weeks 1 to 52 | COMPLETED | Randomized Post-Treatment Follow-up Period: Placebo | 0 | |
| RT Period: Weeks 1 to 52 | COMPLETED | Randomized Post-Treatment Follow-up Period: Volanesorsen | 0 |
1 to 10 of 90Source ClinicalTrials.gov
See all 90 participant flowAdverse events1,368
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 | Urinary tract infection | Infections and infestations | Randomized Treatment Period: Placebo | 0 | 19 | |
| other | Urinary tract infection | Infections and infestations | Randomized Treatment Period: Volanesorsen | 0 | 21 | |
| other | Urinary tract infection | Infections and infestations | Randomized Post-Treatment Follow-up: Placebo | 0 | 7 | |
| other | Urinary tract infection | Infections and infestations | Randomized Post-Treatment Follow-up: Volanesorsen | 2 | 9 | |
| other | Urinary tract infection | Infections and infestations | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | 2 | 12 | |
| other | Urinary tract infection | Infections and infestations | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen | 1 | 12 | |
| other | Sinusitis | Infections and infestations | Randomized Treatment Period: Placebo | 0 | 19 | |
| other | Sinusitis | Infections and infestations | Randomized Treatment Period: Volanesorsen | 0 | 21 | |
| other | Sinusitis | Infections and infestations | Randomized Post-Treatment Follow-up: Placebo | 2 | 7 | |
| other | Sinusitis | Infections and infestations | Randomized Post-Treatment Follow-up: Volanesorsen | 0 | 9 |
1 to 10 of 1,368Source ClinicalTrials.gov
See all 1,368 adverse eventsAdverse event totals18
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Randomized Treatment Period: Placebo | 0 | 19 |
| Total, all-cause mortality | Randomized Treatment Period: Volanesorsen | 0 | 21 |
| Total, all-cause mortality | Randomized Post-Treatment Follow-up: Placebo | 0 | 7 |
| Total, all-cause mortality | Randomized Post-Treatment Follow-up: Volanesorsen | 0 | 9 |
| Total, all-cause mortality | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Placebo/Volanesorsen | 0 | 12 |
| Total, all-cause mortality | Open-Label Extension Period (OLE and OLE Post-Treatment Follow-up): Volanesorsen/Volanesorsen | 0 | 12 |
| Total, other adverse events | Randomized Treatment Period: Placebo | 18 | 19 |
| Total, other adverse events | Randomized Treatment Period: Volanesorsen | 21 | 21 |
| Total, other adverse events | Randomized Post-Treatment Follow-up: Placebo | 4 | 7 |
| Total, other adverse events | Randomized Post-Treatment Follow-up: Volanesorsen | 5 | 9 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37164837 | DERIVED |
Source PubMed
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.
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Read this trial from software.
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