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

• Status: Terminated
• Trial end date: 2019-12-30
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Retrophin, Inc.; Travere Therapeutics, Inc.

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.