Find out more about clinical trials by downloading useful leaflets here
About Ataxia
Clinical trials
Current trials for the ataxias
Friedreich's ataxia
Idebenone (phase III UK/Europe/USA) - see latest press release for details of the European study.
Idebenone withdrawal study (PROTI, Europe – for people who are on the MICONOS extension study)
Varenicline (US) - trial terminated - see press release for details
EGb761 (France)
Pioglitazone (France)
Iron Chelators (deferipron
e) (phase II, Europe)
Resveratrol (Phase I/II, Australia)
EPI-743 for Mitochondrial Respiratory Chain Diseases, including FA (US). See press release for more details
Other ataxias
CoQ10 in mitochondrial diseases (US)
Varenicline in SCA3 (US)
Lithium in SCA2 (phase II, Italy)
Lithium in SCA3 (phase II, Brazil)
Sodium Phenylbutyrate in SCA3 (Brazil)
Riluzole in hereditary CA (Italy)
Intravenous immune globulin Friedreich's ataxia and SCA (phase II, US)
Exercise training programme for CA (US)
Memantine in Fragile X-Associated Tremor/Ataxia Syndrome (US)
For more information on current trials visit http://controlled-trials.com/mrct/
What is a clinical trial?
Clinical trials are an essential part of health research to test whether
a treatment or prevention measure is safe and effective for use in
people.
Trials are carried out following a strict protocol which has to be
approved by an independent ethics committee before the trial begins to
ensure that it is ethical and the patient’s rights will be protected.
A clinical trial often tests a new or experimental treatment against a
placebo; an inactive pill, liquid or other substance that resembles the
drug being tested but has no treatment value. People who participate in
the trial may be divided into groups and some will receive the treatment
being tested and others receive the placebo. This is important to
decide whether the treatment is truly having an effect or if any changes
observed are due to other factors. When both the researchers and
participants involved in the trial are unaware whether they are
receiving a placebo or the active treatment it is called a
‘double-blind’ trial.
Many trials are funded by pharmaceutical companies as they need to test
their drug products in order to gain a license for the drug to be sold
for use in the general public. As researchers identify new potential
treatments for the ataxias, collaborations with pharmaceutical companies
may be increasingly common as they provide the funding to carry out
extensive trials and manufacture the eventual product which will be used
by patients.
Types of Clinical Trials
Pilot study
Very small study carried out to see if it would be worth doing a larger
study and what problems might arise. A pilot study might last a few
weeks to a few months.
Phase I
A small group of around 20-80 people, given the treatment in escalating
doses, in order to evaluate its safety and to identify side effects and
the best dose to use.
Phase II
The treatment is given to a larger group of people, around 100-300, over
a longer period of time (up to 2 years) to further evaluate its safety
and look for beneficial effects.
Phase III
Large trial with 100’s-1000’s of patients taking either the drug or a
placebo substance over a long period of time (3-5 years) in order to
prove whether the treatment is effective and compare it to other
treatments.
Phase IV
Open drug use- when the drug is on the market and available to a much
wider population additional information on its side effects, benefits,
and optimal use might emerge from continuing studies.
Page updated July 2011
