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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
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.
Age
18 - No limit years
Sex
ALL
Healthy Volunteers
No
IONIS Investigative Site
Ann Arbor, Michigan, United States
IONIS Investigative Site
Rochester, Minnesota, United States
IONIS Investigative Site
St Louis, Missouri, United States
IONIS Investigative Site
Morehead City, North Carolina, United States
IONIS Investigative Site
Philadelphia, Pennsylvania, United States
IONIS Investigative Site
Dallas, Texas, United States
IONIS Investigative Site
Leuven, Belgium
IONIS Investigative Site
Rio de Janeiro, Brazil
IONIS Investigative Site
Halifax, Nova Scotia, Canada
IONIS Investigative Site
Münster, Germany
Start Date
December 28, 2015
Primary Completion Date
June 30, 2018
Completion Date
November 13, 2019
Last Updated
October 18, 2021
40
ACTUAL participants
volanesorsen
DRUG
Placebo
DRUG
Lead Sponsor
Akcea Therapeutics
Collaborators
NCT06679270
NCT02654977
Data Source & Attribution
This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.
Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.
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