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

• ClinicalTrials.gov Identifier: NCT01303406
• Recruitment Status: Completed
• First Posted: February 24, 2011
• Results First Posted: March 9, 2016
• Last Update Posted: March 9, 2016
• Sponsor: Santhera Pharmaceuticals
• Information provided by (Responsible Party): Santhera Pharmaceuticals

Study Description

Brief 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.

Condition or disease	Intervention/treatment	Phase
Friedreich's Ataxia	Drug: Idebenone; Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 29 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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
• Study Start Date: April 2011
• Actual Primary Completion Date: July 2012
• Actual Study Completion Date: July 2012

Arms and Interventions

Arm	Intervention/treatment
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	Drug: Placebo
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	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).; Other Name: Catena (approved name in Canada)

Outcome Measures

Primary Outcome Measures :

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 Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 10 Years and older (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

Austria

Innsbruck, Austria

Germany

Bonn, Germany

München, Germany

Tübingen, Germany

Netherlands

Groningen, Netherlands

United Kingdom

The National Hospital, University College London

London, United Kingdom, WC 1N 3BG

Sponsors and Collaborators

Santhera Pharmaceuticals

Investigators

• Principal Investigator: Paola Giunti, M.D; Institute of Neurology, The National Hospital, University College London

More Information

• Responsible Party: Santhera Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT01303406
• Other Study ID Numbers: SNT-III-004
• First Posted: February 24, 2011
• Results First Posted: March 9, 2016
• Last Update Posted: March 9, 2016
• Last Verified: February 2016

Keywords provided by Santhera Pharmaceuticals:

randomized withdrawal; idebenone; friedreich's ataxia; Miconos; Patient reported outcome; ICARS

Additional relevant MeSH terms:

Idebenone; Ataxia; Cerebellar Ataxia; Friedreich Ataxia; Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Mitochondrial Diseases; Metabolic Diseases; Antioxidants; Molecular Mechanisms of Pharmacological Action; Protective Agents; Physiological Effects of Drugs