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Phase 3 N=200 Treatment

Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension)

Freidreich's Ataxia

Enrolled (actual)
200
Serious AEs
24.5%
Results posted
Mar 2018
Primary outcome: Primary: Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs) — 188; 109; 8; 54 participants

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
idebenone (Drug)
Age
Pediatric, Adult, Older Adult · 9+ yrs
Sex
All
Sponsor
Santhera Pharmaceuticals
Primary completion
Jun 2012

Outcome Measures

OutcomeResultp-value
PRIMARY
Measures of Safety and Tolerability: Nature and Frequency of Adverse Events (AEs)
188; 109; 8; 54; 1; 48
PRIMARY
Absolute Change in The International Cooperative Ataxia Rating Scale (ICARS)
1.41; 2.88

Summary

This is an Extension study of the MICONOS main randomised placebo-controlled trial (NCT00905268), and open to those patients completing the main study. The scientific aim of this extension study is to monitor safety and tolerability of idebenone over two years in patients with Friedreich's Ataxia.

Eligibility Criteria

Inclusion Criteria

  • Completion of 52 weeks in study SNT-III-001
  • Body weight ≥ 25 kg
  • Negative urine pregnancy test
  • Eligibility to participate in the present extension study as confirmed by investigator

Exclusion Criteria

  • Safety or tolerability issues arising during the course of SNT-III-001 which in the opinion of the investigator preclude further treatment with idebenone
  • Clinically significant abnormalities of haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of SGOT, SGPT or creatinine
  • Parallel participation in another clinical drug trial
  • Pregnancy or breast-feeding
  • Abuse of drugs or alcohol
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00993967). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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