Who we are
At the Clinical Trial Unit for Paediatrics and Adolescent Medicine, we bridge the gap between research and patient care. Our goal is to provide children and adolescents living with serious or rare diseases with access to new treatment opportunities. We achieve this by conducting clinical trials of investigational medicines that are not yet available on the market.
For many children and adolescents living with serious or rare diseases, there are currently few or no approved treatment options available. In these situations, participation in a clinical trial may provide an important opportunity. Through our clinical studies, patients can gain access to innovative and promising medicines that would otherwise not be available to them. While the full effects of a treatment cannot be known in advance, participation can provide families with a concrete treatment option here and now and may, in some cases, represent the only available therapeutic alternative.
At the same time, the research conducted through these trials contributes to the development of future treatments for children and adolescents. Our aim is to generate new knowledge that can lead to improved treatment options, enhanced quality of life, and better health outcomes for children living with serious or rare diseases.
Our research
Our work ranges from study planning and start-up activities to conducting patient visits, data collection, and follow-up throughout the study period. We conduct both sponsor-initiated and investigator-initiated studies, including clinical drug trials, registry studies and medical device studies.
In our clinical drug trials, we work across a range of disease areas affecting children and adolescents, including skeletal dysplasia, metabolic and lysosomal disorders, neurological diseases, kidney diseases, and other rare genetic diseases.
Through our research, we help generate new knowledge about the safety and effectiveness of medicines for children and adolescents. Today, only 30% of all medicines have been specifically tested and approved for use in children. Therefore, we not only investigate new medicines but also study existing treatments that are already used, or have the potential to be used, in children but have not been approved for a particular disease or patient population. This research provides essential evidence that supports the approval of new treatments and helps make them available to patients, both in Denmark and internationally.
Our research areas continue to evolve through new projects and collaborations.
Learn more about our studies and research areas
Collaborations across the globe
As a Clinical Trial Unit within the Juliane Marie Centre, we collaborate with hospitals, research institutions, patient organisations, and pharmaceutical companies in Denmark and around the world. Through these partnerships, we help ensure that children and adolescents have the opportunity to participate in clinical research of the highest scientific standard and benefit from the latest advances in their disease area.
Many of the conditions we work with are rare, meaning that only a limited number of patients are affected in any one country. To conduct clinical trials and generate meaningful new knowledge, we work closely with other specialised centres across international borders. Through these collaborations, we combine expertise and experience from around the world to support the development of new treatment options for children and adolescents.
Our international network also enables our patients to participate in global clinical studies, while allowing patients from other countries to take part in clinical trials conducted at our unit.