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Eligibility
for people ages 6–65
Location
at Oakland, California and other locations
Dates
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 Neurodegeneration PKAN

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

  • Emory University accepting new patients
    Decatur, Georgia, 30033, United States
  • Children's Hospital of Pittsburgh accepting new patients
    Pittsburgh, Pennsylvania, 15224, United States
  • Massachusetts General Hospital accepting new patients
    Boston, Massachusetts, 02114, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Retrophin, Inc.
Links
Study website
ID
NCT03041116
Phase
Phase 3
Study Type
Interventional
Last Updated
June 1, 2017
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