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

Purpose

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	Intervention	Phase
Friedreich's Ataxia	Drug: Idebenone	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, 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

Resource links provided by NLM:

Genetics Home Reference related topics: ataxia neuropathy spectrum childhood myocerebrohepatopathy spectrum deoxyguanosine kinase deficiency Friedreich ataxia infantile-onset spinocerebellar ataxia Marinesco-Sjögren syndrome mitochondrial neurogastrointestinal encephalopathy disease myoclonic epilepsy myopathy sensory ataxia spinocerebellar ataxia type 1 spinocerebellar ataxia type 2 spinocerebellar ataxia type 3 spinocerebellar ataxia type 6

MedlinePlus related topics: Friedreich's Ataxia

U.S. FDA Resources

Further study details as provided by Santhera Pharmaceuticals:

Primary Outcome 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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Comparison of the proportions of patients 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) ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	80
Study Start Date:	April 2011
Study Completion Date:	July 2012
Primary Completion Date:	July 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
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: 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)
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)

Arms

Assigned Interventions

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: 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)

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)

Eligibility

Ages Eligible for Study:	10 Years and older
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT01303406

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

No publications provided

Responsible Party:	Santhera Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT01303406 History of Changes
Other Study ID Numbers:	SNT-III-004
Study First Received:	February 22, 2011
Last Updated:	August 28, 2012
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 Agency United Kingdom: National Institute for Health Research United Kingdom: Research Ethics Committee Germany: Federal Institute for Drugs and Medical Devices Germany: Ethics Commission Austria: Agency for Health and Food Safety Austria: Ethikkommission

Keywords provided by Santhera Pharmaceuticals:

randomized withdrawal
idebenone
friedreich's ataxia

Miconos
Patient reported outcome
ICARS

Additional relevant MeSH terms:

Ataxia
Friedreich Ataxia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinocerebellar Degenerations
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases

Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Idebenone
Antioxidants
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Protective Agents
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on May 23, 2013