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Clinical trials

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.

Clinical trials are further explained in a useful leaflet from the UK Clinical Research Collaboration, available here



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.


Current trials for the ataxias

Idebenone for Friedreich’s ataxia (phase III UK/Europe/USA)

Erythropoietin (EPO) (phase I/II USA)

Iron Chelators (deferiprone) (phase I/II UK/Europe)

Immune-modifiying therapy in sporadic ataxia (pilot USA)

Lithium for SCA1 (phase I USA)

Riluzole for cerebellar ataxia (phase II Italy)

Valproic acid for SCA3 (phase I China)




Page created May 2008

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