High dose Coenzyme Q10 and vitamin E therapy in Friedreich's ataxia

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Prof Anthony Schapira
Scientific

Department of Clinical Neurosciences
Royal Free & University College Medical School
Rowland Hill Street
London
NW3 2PF
United Kingdom

Primary study design	Interventional
Study design	Randomised double blind controlled trial, involving 2 arms; high doses of coenzyme Q10 / vitamin E; low doses of coenzyme Q10 / vitamin E
Secondary study design	Randomised controlled trial
Scientific title
Study acronym	QES4
Study objectives	High doses of coenzyme Q10 and vitamin E given daily over two years will modifiy disease progression in patients with genetically proven Friedreich's ataxia.
Ethics approval(s)	The Royal Free Local Research Ethics Committee (UK), Ref 5405, approved 16/05/2001.
Health condition(s) or problem(s) studied	Friedreich's ataxia
Intervention	High doses of coenzyme Q10 / vitamin E vs Low doses of coenzyme Q10 / vitamin E
Intervention type	Supplement
Primary outcome measure(s)	Significant improvement in the rate of change in the International Cooperative Ataxia Ratings Scale (ICARS) scores over two years of the trial in the high dose versus the low dose treatment groups
Key secondary outcome measure(s)	1. Rate of change in the kinetic, posture and gait, speech, ocular subscores of the ICARS 2. Rate of change in the following: a. speech tests (PaTa repetition, speed of reading) b. upper limb coordination tests (hand clicker, modified peg board, hand target, BRAIN kinesia) c. lower limb coordination tests (foot target, box) d. cardiac tests (IVS, PWd and fraction shortening) e. activities of daily living scale
Completion date	01/05/2004

Participant type(s)	Patient
Age group	Not Specified
Sex
Target sample size at registration	50
Key inclusion criteria	1. Homozygous for the GAA expansion in the FRDA gene characteristic of the disease 2. Able and willing to attend the assessments over the trial period 3. Over 10 years of age at the start of the trial
Key exclusion criteria	1. Planned surgery during the trial period 2. An ICARS score greater than 80% of the maximum 3. Taking coenzyme Q10, vitamin E or other antioxidants 4. Planned pregnancy
Date of first enrolment	01/09/2001
Date of final enrolment	01/05/2004

Department of Clinical Neurosciences

London
NW3 2PF
United Kingdom

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary
IPD sharing plan

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	01/12/2008		Yes	No