The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy
Summary
The purpose of this study is to evaluate the efficacy and safety of volanesorsen given for 52 weeks in a randomized treatment (RT) period in participants with familial partial lipodystrophy (FPL). Following the randomized treatment period, participants who did not enter the open-label extension (OLE) period went straight to the 13-week post-treatment (PT) follow-up period and participants who were entered in the OLE period continued to receive volanesorsen for another 52 weeks (Weeks 53 to 104). Following the Week 104 visit of the OLE period, participants had an option of continued dosing for up to an additional 52 weeks (Week 105 to 156). Participants who did not enter the OLE period went straight to a 13-week post-treatment follow-up period. Following the Week 104 OLE period, participants were entered a 13-week post-treatment follow-up period, if they did not choose the option for continued dosing.
Timeline
- Start
- 2015-12-28
- Primary completion
- 2018-06-30
- Completion
- 2019-11-13
Outcome
Met primary endpoint
Stopped: “Study terminated early at a time point when sufficient data had been accumulated to inform a decision on further development of volanesoresen in participants with FPL.”
registry analysis (superiority test); Randomized Treatment Period: Placebo vs Randomized Treatment Period: Volanesorsen; p = 0.0009; Difference in Least Square Mean -66.83 (95% CI -104.17 to -29.48); ANCOVA NCT02527343 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Volanesorsen | Antisense oligonucleotide | 300 mg | Subcutaneous |