TerminatedPhase 3

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

Stopped: The study was terminated since fosmetpantotenate did not show statistically significant effects or clinical benefits during the double-blind period.

United States84 participantsStarted 2017-07-17

Plain-language summary

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

Who can participate

Age range6 Years – 65 Years

SexALL

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Inclusion criteria

✓. 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.

Exclusion criteria

✕. 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.

What they're measuring

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

Timeframe: Baseline (Day -1), Week 24

Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period

Timeframe: From Screening until end of Week 24

Trial details

NCT IDNCT03041116

SponsorTravere Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2019-12-30

Results submitted2020-12-02

View on ClinicalTrials.gov More Pantothenate Kinase-Associated Neurodegeneration trials