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

Phase 3 Terminated 84 enrolled Travere Therapeutics, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

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

Timeline

Start: 2017-07-17
Primary completion: 2019-12-30
Completion: 2019-12-30

Outcome

Missed primary endpoint

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

registry analysis (superiority test); Fosmetpantotenate vs Placebo; p =0.9115; Difference in LS Mean -0.1 (95% CI -1.69 to 1.51) NCT03041116 ↗

Publications

Results Klopstock T, Escolar ML, Marshall RD, Perez-Duenas B, Tuller S, Videnovic A, Greblikas F. The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy outcome in patients with pantothenate kinase-associated neurodegeneration. Clin Trials. 2019 Aug;16(4):410-418. doi: 10.1177/1740774519845673. Epub 2019 May 6.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Fosmetpantotenate	Unknown	—	—