Condition category
Nervous System Diseases
Date applied
10/01/2007
Date assigned
01/02/2007
Last edited
27/09/2011
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Prof Anthony Schapira

ORCID ID

Contact details

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

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 measures

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

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

Sponsor information

Organisation

Royal Free Hospital Trust (UK)

Sponsor details

Royal Free Hospital Trust
Pond Street
London
NW3 2PF
United Kingdom

Sponsor type

Government

Website

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

Results - basic reporting

Publication summary

2008 results in http://www.ncbi.nlm.nih.gov/pubmed/19049556

Publication citations

  1. 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.

Additional files

Editorial Notes