Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate Replacement Therapy, in Patients With Pantothenate Kinase-Associated Neurodegeneration (PKAN): A Randomized, Double-Blind, Placebo-Controlled Study With an Open-Label Extension

HS 2018-4223
Movement disorders
Neal Hermanowicz, MD
UCI Main Campus (Hewitt Hall & Gottschalk) and UC Irvine Medical Center
The purpose of this study is to see if fosmetpantotenate improves the symptoms of people with PKAN.

You can participate in this study if you:

  • Are between 6 to 65 years old
  • Have a diagnosis of PKAN by genetic testing (a test that examines DNA [deoxyribonucleic acid] to identify changes in chromosomes, genes, or proteins)
  • Agree to use medically acceptable method of contraception for the duration of the study and for at least 30 days after the last dose of investigational product

You cannot participate in this study if you:

  • Have not been diagnosed with PKAN previously by genetic testing (a test that examines DNA [deoxyribonucleic acid] to identify changes in chromosomes, genes, or proteins)
  • Have needed ventilator support during the last 6 months
  • Have been or are currently enrolled in another clinical study within the 30 days prior to being randomly assigned to treatment
  • Test positive for hepatitis B virus (HBV), human immunodeficiency virus (HIV) and hepatitis C virus (HCV) (markers of certain viral infections for AIDS and hepatitis). If you are diagnosed during the screening period of this study with a reportable disease, the study doctor will be obligated to report this according to regulatory requirements.
  • Have a medical history of certain types of cancer, certain psychiatric conditions, drug or alcohol use disorder, or any suicide attempts in the past year
  • Have had major surgery within 30 days prior to the screening visit
  • Had a deep brain stimulation device implanted within 6 months prior to the screening visit
  • Have abnormal laboratory tests at the screening visit
  • Are a female who is pregnant or breastfeeding or have a positive pregnancy test
  • Have taken deferiprone within 30 days prior to the screening visit
(Note: To be eligible for the study, if you are taking deferiprone, you must agree to a 30-day “washout” period [when you stop taking deferiprone], after you sign a separate consent.)
In total, participation in the study for up to 120 weeks, with 24 weeks in the double-blind randomized period and 96 weeks in the open-label period
Fosmetpantotenate may or may not help PKAN symptoms by improving your muscle contractions (tightening of muscle) or dystonia (involuntary muscle tightening that causes slow repetitive movements or abnormal postures), quality of life, and functional abilities. You will be given medical attention from the study staff during the time you are involved in the study. You may receive information about your health from physical examinations and medical tests done in this study.
You will not be paid for taking part in this study. You may have some expenses from being in the study, such as travel expenses, hotel bills, bus fare or parking fees when you visit the study center. You will be reimbursed for these expenses. 
Fosmetpantotenate (RE-024)
Drug therapy
Breana Chew, Neurology Clinical Trials Unit