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

Stopped early · Phase 3 · Has a placebo group

Conditions studied: Pantothenate Kinase-Associated Neurodegeneration

In brief

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

Key facts

Study ID

NCT03041116

Run by

Travere Therapeutics, Inc.

People needed

84

Starts

2017-07-17

Expected to finish

2019-12-30

Last updated by the study team

2021-01-26

Who can join

Age: 6 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• The participant had required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
• The participant had a deep brain stimulation device implanted within 6 months prior to screening.
• The participant had taken deferiprone within 30 days prior to screening.
• The participant was unable to maintain stable doses of allowed concomitant medications for the first 24 weeks of the study.

Where it is running

• Travere Investigational Site — Irvine, California, United States
• Travere Investigational Site — Washington D.C., District of Columbia, United States
• Travere Investigational Site — Miami, Florida, United States
• Travere Investigational Site — Decatur, Georgia, United States
• Travere Investigational Site — Chicago, Illinois, United States
• Travere Investigational Site — Boston, Massachusetts, United States
• Travere Investigational Site — New York, New York, United States
• Travere Investigational Site — Pittsburgh, Pennsylvania, United States
• Travere Investigational Site — Houston, Texas, United States
• Travere Investigational Site — Toronto, Ontario, Canada
• Travere Investigational Site — Toronto, Ontario, Canada
• Travere Investigational Site — Prague, NAP, Czechia
• Travere Investigational Site — Montpellier, Languedoc-Rousillon, France
• Travere Investigational Site — Paris, Île-de-France Region, France
• Travere Investigational Site — München, Bavaria, Germany
• Travere Investigational Site — Milan, Italy
• Travere Investigational Site — Oslo, Norway
• Travere Investigational Site — Warsaw, Poland
• Travere Investigational Site — Barcelona, Catalonia, Spain
• Travere Investigational Site — Barcelona, Spain

Full record on ClinicalTrials.gov

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