Clinical Trial: Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

Brief Summary: This study is meant to assess the safety and tolerability of idebenone in patients with Friedreich's Ataxia over a 12 months period.

Detailed Summary: The study involves 6 clinic visits.
Sponsor: Santhera Pharmaceuticals

Current Primary Outcome: Change in ICARS [ Time Frame: baseline and 12 months ]

International Cooperative Ataxia Rating Scale (ICARS):

ICARS consists of a one-hundred-point semi-quantitative scale based upon 19 simple testing manoeuvres compartmentalized into postural and stance disorders, limb ataxia, dysarthria and oculomotor disorders and has been previously used in this patient population with good inter-rater reliability.

Scores for each subscale quantify the extent of ataxia in each clinically important area. Subscale scores are summed to give a total score ranging from 0 (best) to 100 (worst).



Original Primary Outcome:

  • Hematology [ Time Frame: 12 months ]
  • Nature and frequency of AEs [ Time Frame: 12 months ]
  • Blood and urine chemistry [ Time Frame: 12 months ]
  • Physical/neurological examination and vital signs [ Time Frame: 12 months ]
  • Electrocardiograms (ECGs) [ Time Frame: 12 months ]


Current Secondary Outcome:

  • FARS (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ]
  • Nature and Frequency of Adverse Events [ Time Frame: 12 Months ]


Original Secondary Outcome:

  • FARS [ Time Frame: 12 Months ]
  • ICARS [ Time Frame: 12 Months ]
  • FACT [ Time Frame: 12 Months ]


Information By: Santhera Pharmaceuticals

Dates:
Date Received: June 11, 2008
Date Started: July 2008
Date Completion:
Last Updated: May 24, 2013
Last Verified: May 2013