A REVIEW ON FRIEDREICH ATAXIA, A NEURODEGENERATIVE DISORDER

  • DHRUVKUMAR MITESHKUMAR SONI Bombay College of Pharmacy, University of Mumbai.

Abstract

The present report discusses about the clinical trial requirements for Friedreich ataxia disease such as inclusion and exclusion criteria for the patients,primary/secondary outcome measuresrequired for assessing the efficacy of therapies viarating scales, physical activity tests, biomarkersand number of subjects/patients to be enrolled in each phase of the clinical trials of Friedreich ataxia (F.A.). These parameters have been selected from the ongoing and completed clinical trials for F.A. as reported in Clinical trials.gov and Friedreich Ataxia Research Alliance (FARA) official website. A total of 33 clinical trialdetails werereviewed for compilation of the above mentioned parameters. For the purpose of this report, the most useful and most commonly assessedprimary/secondary outcome measureswith respect to efficacy as well as inclusion and exclusion criteria have been selected and are being cited in this review.The outcome measures for the purpose of efficacy trials can broadly be classified as primary and secondaryoutcome measures and in each category, subclasses are being presented in the order of their priority as determined by their usefulness and common occurrence in all trials mentioned. These outcome measuresincludeRating Scales for Friedreich Ataxia, Physical Activity Tests and Biomarkers, evaluation of Cardiac functionality viz. left ventricular wall mass, non-invasive measures of systolic and diastolic ventricular function, echocardiography and immunogenicity.The genetic predisposition of Friedreich ataxia (epigenetics) leading to the pathogenesis of the disease is also discussed in this report. Based on above primary and secondary outcomes measures and inclusion and exclusion criteria, a tabular representation of the ongoing/ completed clinical trials representing the efficacy studies of the test molecules and the patient involvements in each clinical trial is also been listed. Various aspects of clinical trialsneeded for assessing ataxic stage in Friedreich ataxia disease is been reviewed in this article.

Key words:Friedreich ataxia, Friedreich Ataxia Research Alliance (FARA), Inclusion and Exclusion criteria, primary and secondary outcome measures, rating scales, biomarkers, physical activity tests.

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Author Biography

DHRUVKUMAR MITESHKUMAR SONI, Bombay College of Pharmacy, University of Mumbai.

MASTER OF PHARMACY GRADUATE SPECIALIZING IN PHARMACEUTICS FROM BOMBAY COLLEGE OF PHARMACY, UNIVERSITY OF MUMBAI, ATTAINED FIRST CLASS WITH GRADE "A".

ATTAINED FIRST RANK IN ALL SEMESTERS OF BACHELOR OF PHARMACY, V.M.H.P.SHAH COLLEGE OF PHARMACY, UNIVERSITY OF MUMBAI

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1.
SONI D. A REVIEW ON FRIEDREICH ATAXIA, A NEURODEGENERATIVE DISORDER. JDDT [Internet]. 15Nov.2014 [cited 31Mar.2020];4(6):45-2. Available from: http://jddtonline.info/index.php/jddt/article/view/1013