NCT02527343

The BROADEN Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Partial Lipodystrophy

A Randomized, Double-Blind, Placebo-Controlled, With an Open Label Extension, Phase 2/3 Study of ISIS 304801 Administered Subcutaneously to Patients With Familial Partial Lipodystrophy

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 40 (actual)
• Sponsor: Akcea Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Familial Partial Lipodystrophy

Interventions

Type	Name	Description
DRUG	volanesorsen	300 mg of volanesorsen administered subcutaneous (SC) injection, once-weekly (QW).
DRUG	Placebo	Volanesorsen-matching placebo administered SC injection.

Timeline

• Start date: 2015-12-28
• Primary completion: 2018-06-30
• Completion: 2019-11-13
• First posted: 2015-08-19
• Last updated: 2021-10-18
• Results posted: 2021-10-18

Locations

12 sites across 7 countries: United States, Belgium, Brazil, Canada, Germany, Netherlands, Russia

Regulatory

• FDA-regulated drug study