Brief Title
Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants
Official Title
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
Brief Summary
This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period
Secondary Outcome
Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period
Condition
Pantothenate Kinase-Associated Neurodegeneration
Intervention
Fosmetpantotenate
Study Arms / Comparison Groups
Fosmetpantotenate
Description: Administered as powder for reconstitution.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
84
Start Date
July 17, 2017
Completion Date
December 30, 2019
Primary Completion Date
December 30, 2019
Eligibility Criteria
Inclusion Criteria: 1. The participant had a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 gene. 2. The participant was male or female aged 6 to 65 years, inclusive. 3. The participant had a score of ≥ 6 on the PKAN-specific activities of daily living measure. Exclusion Criteria: 1. The participant had required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization. 2. The participant had a deep brain stimulation device implanted within 6 months prior to screening. 3. The participant had taken deferiprone within 30 days prior to screening. 4. The participant was unable to maintain stable doses of allowed concomitant medications for the first 24 weeks of the study.
Gender
All
Ages
6 Years - 65 Years
Accepts Healthy Volunteers
No
Contacts
Thomas Klopstock, MD, ,
Location Countries
Canada
Location Countries
Canada
Administrative Informations
NCT ID
NCT03041116
Organization ID
024PKAN15004
Responsible Party
Sponsor
Study Sponsor
Travere Therapeutics, Inc.
Study Sponsor
Thomas Klopstock, MD, Principal Investigator, Klinikum der Universität München
Verification Date
January 2021