Summary

for people ages 6-65 (full criteria)
at Irvine, California and other locations
study started
estimated completion

Description

Summary

This study will investigate whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, is safe and effective in treating patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN).

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

Keywords

Pantothenate Kinase-Associated NeurodegenerationPKANNerve DegenerationPantothenic Acidfosmetpantotenate (RE-024)

Eligibility

You can join if…

Open to people ages 6-65

  1. The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene.
  2. The patient is male or female aged 6 to 65 years, inclusive.
  3. The patient has a score of ≥ 6 on the PKAN-specific activities of daily living measure (PKAN-ADL)

You CAN'T join if...

  1. The patient has required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
  2. The patient has had a deep brain stimulation (DBS) device implanted within 6 months prior to screening.
  3. The patient has taken deferiprone within 30 days prior to screening.
  4. The patient is unable to maintain stable doses of allowed concomitant medications for the first 24 weeks of the study.

Locations

  • University of California Irvine, Clinical Trials Unit
    IrvineCalifornia92697United States
  • The University of Texas Health Science Center at Houston
    HoustonTexas77030United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Retrophin, Inc.
Links
Study website
ID
NCT03041116
Phase
Phase 3
Study Type
Interventional
Last Updated