An efficacy,safety and tolerability study of Fosmetpantotenate in PKAN
Research type
Research Study
Full title
EFFICACY, SAFETY, AND TOLERABILITY OF FOSMETPANTOTENATE (RE-024), A PHOSPHOPANTOTHENATE REPLACEMENT THERAPY, IN PATIENTS WITH PANTOTHENATE KINASE-ASSOCIATED NEURODEGENERATION (PKAN): A RANDOMISED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDY WITH AN OPEN-LABEL EXTENSION
IRAS ID
217001
Contact name
Sukhdip Rai
Contact email
Sponsor organisation
Retrophin Inc
Eudract number
2016-001955-29
Duration of Study in the UK
3 years, 1 months, 1 days
Research summary
This study is sponsored by Retrophin, Inc. and the name of this investigational new drug is Fosmetpantotenate (RE-024).
PKAN patients with this condition can have abnormal muscle movements, twitching, spasms, rigidity, pain, difficulties with speech, difficulty swallowing, and other problems and there is currently no treatment available. The investigational new drug is fosmetpantotenate and the purpose of this study is to see if it works to help people with PKAN. This study will determine whether the study drug fosmetpantotenate is safe to use, and will improve the signs and symptoms of PKAN, and stabilise the progression of PKAN. It will be important to consider how long and how much fosmetpantotenate stays in the blood. There will be one recruiting site in the UK which will be aiming to enrol 4 participants onto this trial. This trial will also be set up in nine other countries.
REC name
London - Chelsea Research Ethics Committee
REC reference
16/LO/2157
Date of REC Opinion
3 Mar 2017
REC opinion
Further Information Favourable Opinion