Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
FAQES4
Study information
Scientific title
Acronym
QES4
Study hypothesis
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
The Royal Free Local Research Ethics Committee (UK), Ref 5405, approved 16/05/2001.
Study design
Randomised double blind controlled trial, involving 2 arms; high doses of coenzyme Q10 / vitamin E; low doses of coenzyme Q10 / vitamin E
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Condition
Friedreich's ataxia
Intervention
High doses of coenzyme Q10 / vitamin E vs Low doses of coenzyme Q10 / vitamin E
Intervention type
Supplement
Phase
Not Specified
Drug names
coenzyme Q10, vitamin E
Primary outcome measure
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
Secondary outcome measures
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
Overall trial start date
01/09/2001
Overall trial end date
01/05/2004
Reason abandoned (if study stopped)
Eligibility
Participant 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
Participant type
Patient
Age group
Not Specified
Gender
Not Specified
Target number of participants
50
Participant 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
Recruitment start date
01/09/2001
Recruitment end date
01/05/2004
Locations
Countries of recruitment
United Kingdom
Trial participating centre
Department of Clinical Neurosciences
London
NW3 2PF
United Kingdom
Funders
Funder type
Charity
Funder name
Ataxia UK (UK)
Alternative name(s)
Funding Body Type
private sector organisation
Funding Body Subtype
other non-profit
Location
United Kingdom
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list
2008 results in http://www.ncbi.nlm.nih.gov/pubmed/19049556
Publication citations
-
Results
Cooper JM, Korlipara LV, Hart PE, Bradley JL, Schapira AH, Coenzyme Q10 and vitamin E deficiency in Friedreich's ataxia: predictor of efficacy of vitamin E and coenzyme Q10 therapy., Eur. J. Neurol., 2008, 15, 12, 1371-1379, doi: 10.1111/j.1468-1331.2008.02318.x.