Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (IONIA)

Tracking Information
First Submitted Date ICMJE	September 7, 2007
First Posted Date ICMJE	October 1, 2007
Results First Submitted Date	June 7, 2011
Results First Posted Date	December 29, 2011
Last Update Posted Date	February 3, 2012
Study Start Date ICMJE	December 2007
Actual Primary Completion Date	April 2009 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: November 22, 2011)	ICARS [ Time Frame: baseline and 6 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 Measures ICMJE; (submitted: September 28, 2007)	ICARS [ Time Frame: 6 months ]
Change History	Complete list of historical versions of study NCT00537680 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: November 22, 2011)	FARS [ Time Frame: 6 Months ]Friedreich's Ataxia Rating Scale; ADL of FARS [ Time Frame: 6 Months ]ADL=Activities of Daily Living; FACT [ Time Frame: 6 Months ]Friedreich's Ataxia Composite Test
Original Secondary Outcome Measures ICMJE; (submitted: September 28, 2007)	FARS [ Time Frame: 6 Months ]; ADL of FARS [ Time Frame: 6 Months ]; FACT [ Time Frame: 6 Months ]
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Official Title ICMJE	A Phase III Double-Blind, Randomized, Placebo-Controlled Study of the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Brief Summary	This study is meant to assess the effectiveness of idebenone on neurological outcome measures in patients with Friedreich's Ataxia over a 6 months period.
Detailed Description	The study involves 6 clinic visits and upon completion the possibility to join a 12 months extension study where all patients will receive high dose Idebenone.
Study Type ICMJE	Interventional
Study Phase	Phase 3
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Friedreich's Ataxia
Intervention ICMJE	Drug: Idebenone Patients ≤ 45 kg/99 lbs: idebenone 450 mg/day; Patients > 45 kg/99 lbs: idebenone 900 mg/day; Drug: Idebenone Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day; Drug: Placebo Placebo was provided as film-coated tablets that were the same size, weight and appearance as the idebenone tablets.
Study Arms	Experimental: 1 mid dose Idebenone Intervention: Drug: Idebenone; Experimental: 2 high dose Idebenone Intervention: Drug: Idebenone; Placebo Comparator: 3 Intervention: Drug: Placebo
Publications *	Meier T, Perlman SL, Rummey C, Coppard NJ, Lynch DR. Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012 Feb;259(2):284-91. doi: 10.1007/s00415-011-6174-y. Epub 2011 Jul 22.; Lagedrost SJ, Sutton MS, Cohen MS, Satou GM, Kaufman BD, Perlman SL, Rummey C, Meier T, Lynch DR. Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011 Mar;161(3):639-645.e1. doi: 10.1016/j.ahj.2010.10.038. Epub 2011 Jan 31.; Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010 Aug;67(8):941-7. doi: 10.1001/archneurol.2010.168.
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Completed
Actual Enrollment ICMJE; (submitted: May 28, 2009)	70
Original Estimated Enrollment ICMJE; (submitted: September 28, 2007)	51
Actual Study Completion Date	April 2009
Actual Primary Completion Date	April 2009 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	Inclusion criteria: Documented diagnosis of Friedreich's Ataxia (FRDA) with confirmed FRDA mutations; Patients 8 - 17 years of age at baseline; Patients with a body weight greater than 25 kg/55 lbs at baseline; Patients able to walk at least 10 meters without accompanying person (ICARS Walking Capacities score ≤6) at screening and baseline; Patients who in the opinion of the investigator are able to comply with the requirements of the study, including swallowing the study medication; Negative urine pregnancy test at screening and baseline (female patients of childbearing potential) Exclusion criteria: Treatment with idebenone, Coenzyme Q10 or vitamin E (if taken at a dose 5 times above the daily requirement) within the past month; Patients with International Cooperative Ataxia Rating Scale (ICARS) score of greater than 54 or less than 10 at screening; Pregnancy and/or breast-feeding; Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal of Aspartate Aminotransferase (AST; also known as GOT), Alanine Aminotransferase (ALT; also known as GPT) or creatinine; History of abuse of drugs or alcohol; Participation in the previous (Phase II) study of idebenone at the National Institutes of Health (NIH); Participation in a trial of another investigational drug within the last 3 months
Sex/Gender	Sexes Eligible for Study: All
Ages	8 Years to 17 Years (Child)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT00537680
Other Study ID Numbers ICMJE	SNT-III-002
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Santhera Pharmaceuticals
Study Sponsor ICMJE	Santhera Pharmaceuticals
Collaborators ICMJE	Not Provided
Investigators ICMJE	Principal Investigator: Susan Perlman, MD University of California, Los Angeles Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia
PRS Account	Santhera Pharmaceuticals
Verification Date	February 2012
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP