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Completed

NCT Number: NCT01303406

Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)

This is a Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study. The aim is to further investigate the effects of idebenone in patients with Friedreich's ataxia.

The objective of the PROTI study is to establish whether patients can correctly determine which treatment assignment (placebo or idebenone) they received during the randomised phase of the trial, and identify any potential changes on symptoms or activities.

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Key information

Conditions

Friedreich's Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Friedreich Ataxia Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Age range

10 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Innsbruck, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Completion of V5 (Month 12), V6 (Month 18), or V7 (Month 24) in the MICONOS extension study
  • Patients who in the opinion of the investigator are able to comply with the requirements of the study
  • Body weight ≥ 25kg
  • Negative urine pregnancy test

Exclusion criteria

  • AE during the course of the MICONOS extension study which in the opinion of the investigator is attributable to idebenone and precludes further treatment with idebenone
  • Clinically significant abnormalities of clinical haematology or biochemistry including, but not limited to, elevations greater than 1.5 times the upper limit of normal SGOT, SGPT or creatinine
  • Parallel participation in another clinical drug trial
  • Pregnancy or breast-feeding
  • Abuse of drugs or alcohol
  • Any change of concomitant medication within the last 30 days that in the opinion of the investigator the intake could negatively impact the study

Treatment and study plan

Idebenone

Drug

All PROTI patients randomised to idebenone treatment will receive high dose idebenone. This is defined according to body weight. In patients weighing 45 kg or less, it is 1350 mg/day (3 x 150 mg tablets three times per day with meals). In patients weighing more than 45 kg, it is 2250 mg/day (5 x 150 mg tablets three times per day with meals).

Other names: Catena (approved name in Canada)

Placebo

Drug

Primary outcomes

  1. Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone

    Time frame: At 2 months after study start

    The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.

Secondary outcomes

  1. Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms

    Time frame: Within 2 months (i.e. Early withdrawal visit)

    There was no Withdrawal due to recurrence or worsening of FRDA symptoms

Sponsors and collaborators

Lead sponsor

Santhera Pharmaceuticals

Industry

Registry information

Official study title

A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone

Acronym: PROTI

Important dates

Study start
2011
Primary completion
2012
Study completion
2012
First posted
Feb 24, 2011
Registry last updated
Mar 9, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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