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

Overview

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.

Full Title of Study: “A Phase IIIb Double-Blind, Randomised, Placebo-Controlled Study of Patient Reported Outcomes in Friedreich’s Ataxia Patients After Withdrawal From Treatment With Idebenone”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: July 2012

Interventions

  • Drug: Idebenone
    • 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).
  • Drug: Placebo

Arms, Groups and Cohorts

  • Placebo Comparator: Placebo
    • Following the body weight, patients will be allocated to one of the following regimen: Placebo Patients < 45 kg – 3 tablets 3 times a day with meals Placebo Patients > 45 kg – 5 tablets 3 times a day with meals
  • Experimental: idebenone
    • Following the body weight, patients will be allocated to one of the following regimen: Idebenone Patients < 45 kg – 3 tablets 3 times a day with meals Idebenone Patients > 45 kg – 5 tablets 3 times a day with meals

Clinical Trial Outcome Measures

Primary Measures

  • 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 Measures

  • 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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 10 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Santhera Pharmaceuticals
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Paola Giunti, M.D, Principal Investigator, Institute of Neurology, The National Hospital, University College London

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