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

NCT01303406 | Completed Phase 3 Results DMC

• 1 other identifier: SNT-III-004
• Study Type: interventional
• Target: 29
• Locations: 4 countries
• Sites: 6

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.

Conditions

Friedreich's Ataxia

Keywords

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

Outcome Measures

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

  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

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

  Within 2 months (i.e. Early withdrawal visit)

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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

idebenone

EXPERIMENTAL

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

Interventions

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).

Also known as: Catena (approved name in Canada)

idebenone

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 10 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (6)

Unknown Facility

Innsbruck, Austria

Location

Unknown Facility

Bonn, Germany

Location

Unknown Facility

München, Germany

Location

Unknown Facility

Tübingen, Germany

Location

Unknown Facility

Groningen, Netherlands

Location

The National Hospital, University College London

London, WC 1N 3BG, United Kingdom

Location

MeSH Terms

Conditions

Friedreich Ataxia

Interventions

idebenone

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Dr Paola Giunti
• Organization: National Hospital for Neurology and Neurosurgery

violet.le-fevre@uclh.nhs.uk

020 3448 3141

Study Officials

• Paola Giunti, M.D

  Institute of Neurology, The National Hospital, University College London

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 22, 2011
• First Posted: February 24, 2011
• Study Start: April 1, 2011
• Primary Completion: July 1, 2012
• Study Completion: July 1, 2012
• Last Updated: March 9, 2016
• Results First Posted: March 9, 2016

Record last verified: 2016-02

Locations

AU (1); DE (3); GB (1); NL (1)