Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Patients: A Randomized, Double-Blind, Placebo-Controlled Study With an Open-Label Extension
Status: Terminated
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Maximum Age: 65
Healthy Volunteers: f
View:
• The participant had a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 gene.
• The participant was male or female aged 6 to 65 years, inclusive.
• The participant had a score of ≥ 6 on the PKAN-specific activities of daily living measure.
Locations
United States
California
Travere Investigational Site
Irvine
Washington, D.c.
Travere Investigational Site
Washington
Florida
Travere Investigational Site
Miami
Georgia
Travere Investigational Site
Decatur
Illinois
Travere Investigational Site
Chicago
Massachusetts
Travere Investigational Site
Boston
New York
Travere Investigational Site
New York
Pennsylvania
Travere Investigational Site
Pittsburgh
Texas
Travere Investigational Site
Houston
Other Locations
Canada
Travere Investigational Site
Toronto
Travere Investigational Site
Toronto
France
Travere Investigational Site
Montpellier
Travere Investigational Site
Paris
Germany
Travere Investigational Site
München
Italy
Travere Investigational Site
Milano
Norway
Travere Investigational Site
Oslo
Poland
Travere Investigational Site
Warsaw
Spain
Travere Investigational Site
Barcelona
Travere Investigational Site
Barcelona
Time Frame
Start Date: 2017-07-17
Completion Date: 2019-12-30
Participants
Target number of participants: 84
Treatments
Experimental: Fosmetpantotenate
Administered as powder for reconstitution.
Placebo_comparator: Placebo
Administered as powder for reconstitution.
Authors
Related Therapeutic Areas
Sponsors
Leads: Travere Therapeutics, Inc.