NCT03041116

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 84 (actual)
• Sponsor: Travere Therapeutics, Inc. · Industry
• Sex: All
• Age: 6 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.

Conditions

• Pantothenate Kinase-Associated Neurodegeneration

Interventions

Type	Name	Description
DRUG	Fosmetpantotenate	Daily dosing
DRUG	Placebo	Daily dosing

Timeline

• Start date: 2017-07-17
• Primary completion: 2019-12-30
• Completion: 2019-12-30
• First posted: 2017-02-02
• Last updated: 2021-01-26
• Results posted: 2020-12-29

Locations

20 sites across 9 countries: United States, Canada, Czechia, France, Germany, Italy, Norway, Poland, Spain

Regulatory

• FDA-regulated drug study