Clinical Research

Is medical research that studies people to understand health conditions better. It ultimately helps to improve the way doctors treat and prevent medical conditions. It allows researchers learn how the body works, how conditions develop in people, which behaviours and treatment help people stay healthy and prevent illness. The aim is to use science to improve people’s health care and health over time. The participants who join and take part in clinical research studies may or may not get an immediate direct benefit. However, their participation in research will ultimately improve the lives of future children living with similar health conditions.

There are two main types of clinical research:

1. Clinical trials/ interventional studies and

2. Observational/ non-interventional studies

1. Clinical Trials

Clinical trials are research studies in which participants get one or more treatments or therapies to test what happens in people. These studies test new methods of screening, prevention, diagnosis, or treatment of a disease. In some clinical trials, researchers randomly assign participants to treatments. This randomisation is vital as it ensures that any positive benefits of the treatment are due to the treatment being tested and not due to other differences in the participants or the participants research experiences. In short it makes research more accurate, reliable, and fair for all.

When you take part in a clinical trial, you add to our knowledge about your health condition and help improve care for people in the future.

There are currently no open clinical trials at NCIMD, although we hope to have one soon.

2. Observational studies

Observational studies are research studies in which researchers simply collect information (called data) from participants (prospective) or look at data that was already collected (retrospective). The data may be about participants’ health, bloods, treatments, procedures, family history etc. In observational studies, researchers do not assign participants to get a treatment. If there is a treatment, participants were already using it as part of their regular health care or daily life.

NCIMD are currently involved in two types of observational studies – registries and natural history studies.

Registries

A patient registry is an organized collection of data that patients agree to give. Researchers can use a patient registry to quickly access data provided by hundreds, or thousands, of similar patients.

Natural History Studies

Natural history studies are a way of collecting information to increase the understanding of a disease and its management. A natural history study is a preplanned, observational study intended to track the course of the disease. Its purpose is to identify demographic, genetic, environmental, and other variables (e.g., treatment modalities, concomitant medications) that relate to the disease's development and outcomes.

NCIMD currently have four externally (Sponsor) funded observational studies in Homocystinuria (HCU)/ Cystathionine beta-synthase deficiency,  Mucopolysaccharidosis type IV (MPS IV)/ Morquio syndrome,  Mucopolysaccharidosis type 1 (MPS 1)/ Scheie and Hurler syndrome.

Each of these studies is led by a principal investigator (PI). At NCIMD the PI is usually a Consultant Metabolic Paediatrician or medical doctor. The PI leads a team of research staff that could include doctors, dieticians, nurses, researchers, and healthcare assistants. The Consultant is qualified and knows the patient well ensuring that patient safety and well-being is precedence.

For more information contact metabolic.research@childrenshealthireland.ie.