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Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients (IONIA-E)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT00697073
First received: June 11, 2008
Last updated: May 24, 2013
Last verified: May 2013

June 11, 2008
May 24, 2013
July 2008
May 2010   (final data collection date for primary outcome measure)
Change in ICARS [ Time Frame: baseline and 12 months ] [ Designated as safety issue: No ]

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).

  • Hematology [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Nature and frequency of AEs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Blood and urine chemistry [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Physical/neurological examination and vital signs [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
  • Electrocardiograms (ECGs) [ Time Frame: 12 months ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00697073 on ClinicalTrials.gov Archive Site
  • FARS (Friedreich's Ataxia Rating Scale) [ Time Frame: baseline and 12 Months ] [ Designated as safety issue: No ]
  • Nature and Frequency of Adverse Events [ Time Frame: 12 Months ] [ Designated as safety issue: Yes ]
  • FARS [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • ICARS [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • FACT [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
A Phase III Open-Label, Single Group Extension Study of the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients

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

The study involves 6 clinic visits.

Interventional
Phase 3
Allocation: Non-Randomized
Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Friedreich's Ataxia
Drug: Idebenone
Patients ≤ 45 kg/99 lbs: idebenone 1350 mg/day; Patients > 45 kg/99 lbs: idebenone 2250 mg/day
Experimental: 1
high dose Idebenone
Intervention: Drug: Idebenone
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
68
May 2010
May 2010   (final data collection date for primary outcome measure)

Inclusion criteria:

  • Friedreich's ataxia patients completing core study SNT-III-002 (NCT00537680) and presenting at Week 24 (Visit 5) of that study
  • Body weight ≥ 25kg/55 lbs
  • Negative urine pregnancy test
  • Patients who in the opinion of the investigator are able to comply with the requirements of this study

Exclusion criteria:

  • Adverse events during the course of SNT-III-002(NCT00537680)which in the opinion of the investigator are attributable to idebenone and preclude further treatment with idebenone
  • Clinically significant abnormalities of clinical hematology or biochemistry including, but not limited to, elevations greater than 2 times the upper limit of normal of AST, ALT or creatinine
  • Treatment with coenzyme Q10, vitamin E (if taken at a dose 5 times above the daily requirement) or other sources of idebenone within the past month
  • Parallel participation in another clinical drug trial
  • Past or present history of abuse of drugs or alcohol
  • Pregnancy or breast-feeding
Both
8 Years to 18 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00697073
SNT-III-002-E
Yes
Santhera Pharmaceuticals
Santhera Pharmaceuticals
Not Provided
Principal Investigator: Susan Perlman, MD University of California, Los Angeles
Principal Investigator: David Lynch, MD Children's Hospital of Philadelphia
Santhera Pharmaceuticals
May 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP