Clinical Trials

New treatment trials in thalassaemia

Aims

Clinical trials are medical research studies aim to find out whether a medical strategy, treatment, or device is safe and effective for humans to use or consume.

Background

All clinical trials of new medicines, or device go through a series of phases to test whether they’re safe and whether they work.
A control will be used to test against the medicines. This will either be a placebo or a standard treatment already in use.
A clinical trial will usually consist of four phases:

Phase 1 trials:

Researchers trial the drug in a small number of individuals (human volunteers) for the first time, who may be healthy volunteers, are given the medicine.
Researchers test for side effects and calculate what the right dose might be to use in treatment.
Researchers start with small doses and only increase the dose if the volunteers do not experience any side effects, or if they only experience minor side effects.

Phase 2 trials:

Ill individuals are chosen in larger group to test the new medicine. This is to get a better idea of its effects in the short term.

Phase 3 trials:

Involve several thousand patients and carried out on medicines that have passed phases 1 and 2.
The medicine is tested in this phase and compared against an existing treatment or a placebo to see if it’s better in practice and if it has important side effects.
Trials often last a year or more.

Phase 4 trials:

The safety, side effects and effectiveness of the medicine continue to be studied while it’s being used in practice. All medicines that have passed all the previous stages and have been given marketing licences (means the medicine is available on prescription) will pass through this phase.

Benefits

In general, clinical studies are designed to add to medical knowledge related to the treatment, diagnosis, and prevention of diseases or conditions. Some common reasons for conducting clinical studies include:

• Evaluating one or more interventions (for example, drugs, medical devices, approaches to surgery or radiation therapy) for treating a disease, syndrome, or condition
• Finding ways to prevent the initial development or recurrence of a disease or condition. These can include medicines, vaccines, or lifestyle changes, among other approaches.
• Evaluating one or more interventions aimed at identifying or diagnosing a particular disease or condition
• Examining methods for identifying a condition or the risk factors for that condition
• Exploring and measuring ways to improve the comfort and quality of life through supportive care for people with a chronic illness.

Luspatercept

Luspatercept

 

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