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

Friedreich's Ataxia Clinical Trial

— PROTI  

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01303406
• Other study ID #: SNT-III-004
• Status: Completed
• Phase: Phase 3
• First received: February 22, 2011
• Last updated: February 10, 2016
• Start date: April 2011
• Est. completion date: July 2012

Study information

• Verified date: February 2016
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)Netherlands: Medical Ethics Review Committee (METC)United Kingdom: Medicines and Healthcare Products Regulatory AgencyUnited Kingdom: National Institute for Health ResearchUnited Kingdom: Research Ethics CommitteeGermany: Federal Institute for Drugs and Medical DevicesGermany: Ethics CommissionAustria: Agency for Health and Food SafetyAustria: Ethikkommission
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 29
• Est. completion date: July 2012
• Est. primary completion date: July 2012
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 10 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia
• Friedreich's Ataxia

Intervention

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).
• Placebo

Locations

Country	Name	City	State
United Kingdom	The National Hospital, University College London	London

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

Austria,  Germany,  Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone	The primary efficacy endpoint was the comparison of the number of patients randomized to idebenone and placebo, who assessed that they received idebenone treatment.	At 2 months after study start	Yes
Secondary	Comparison of the Percentage of Participants Randomised to Idebenone and Placebo Who Withdrew Early Due to Recurrence or Worsening of FRDA Symptoms	There was no Withdrawal due to recurrence or worsening of FRDA symptoms	Within 2 months (i.e. Early withdrawal visit)	Yes