Clinical Trial: Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), A Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Pat
Brief Summary: This study will investigate whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, is safe and effective in treating patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN).
Detailed Summary:
Sponsor: Retrophin, Inc.
Current Primary Outcome:
- Evaluate change in the score of a PKAN specific activities of daily living measure (PKAN-ADL) [ Time Frame: 24 weeks ]
- Evaluate safety and tolerability by occurrence of adverse events classified by MedDRA classification dictionary and safety assessments including vital signs, physical examinations, clinical laboratory tests, C-SSRS assessments, and electrocardiograms. [ Time Frame: 24 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome: Evaluate change in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score [ Time Frame: 24 weeks ]
Original Secondary Outcome: Same as current
Information By: Retrophin, Inc.
Dates:
Date Received: December 2, 2016
Date Started: June 2017
Date Completion: December 2020
Last Updated: May 15, 2017
Last Verified: May 2017
