Clinical Trials Glossary

Here you can find definitions of all the most commonly used words when discussing clinical trials. Think there is a word missing? Drop the research team a message.

ALSFRS-R stands for ALS Functional Rating Scale-Revised. This is a rating scale used by doctors to assess progression and severity of MND. Twelve questions about specific motor functions such as dressing, eating, walking etc are scored by the patient with 0 meaning no function and 4 meaning full function.

An ASO is a short synthetic single strand of DNA that targets a specific region of a gene that produces a faulty protein known to cause disease. It can reduce production of that faulty protein or stop it from being expressed completely, or restore function to that protein.

Any measurable indicator of biological changes. Biomarkers can confirm the presence of disease and are used to monitor disease progression or response to drug treatments.

Neither the researcher nor the participant knows what treatment the participant is receiving.

A programme which allows people living with MND who do not fit the inclusion criteria for a clinical trial to be able to have the treatment.

When both the researcher and the participant know the treatment that the participant is receiving.

An open label extension to a trial is where all participants (including those on the treatment and those on the placebo) are given the treatment.

An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.

The movement of drugs within the body.

A dummy drug which looks, smells and tastes the same as the treatment drug.

Where participants in trials are assigned to the placebo group or treatment group at random. This is usually carried out by computer and prevents bias in choosing which patients get the trial drug.

This is where the drug is delivered by an injection under the skin.

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