A record number of clinical trials taking place at New Children's Hospital

The aim of clinical trials involving children is to develop more effective options for the current treatment methods, and to find new treatments for conditions that have previously been untreatable.  In comparison to recent years, the unit has received significantly more offers for studies, and several new trials are about to begin.

Currently ongoing drug trials include treatments for diabetes, migraine, surgical conditions, and rare diseases. Half of the trials concern cancer medicines. Clinical trials are used to examine drugs, treatments, medical devices, and diagnostic methods to identify various illnesses sooner and to improve care.

An increasing number of child patients can participate in drug trials

New medicines are often first studied in adults, which is why pediatric patients do not benefit from the results as quickly and efficiently as adults. Clinical trials involving children are difficult to arrange, because of their high costs and complexity. However, trials are needed to provide new and better treatments for children.

“The Clinical Trial Unit in New Children's Hospital has expanded significantly in the past few years. We have much better resources to conduct drug and device trials now. An increasing number of children treated in the hospital receive treatment through one of our trials,” explains Head Physician Matti Hero.

HUS has strong potential to attract new clinical drug trials. By Finnish standards, HUS’ population base is large. Extensive national and international co-operation networks facilitate rapid access to the newest and most effective treatments for patients.

Help in rare diseases

When scientific evidence and products suitable for children are not available, children need to be treated in off-label ways. The majority of these patients are also the most vulnerable: newborn babies and children with rare diseases.

About 70% of rare diseases start in childhood, but only about 5% of rare diseases have an officially approved treatment.

“For many patients with a rare disease, participating in a clinical drug trial is the only way to receive targeted medication,” Hero continues.

The treatment of rare diseases requires close collaboration both nationally and internationally. Research projects initiated at HUS benefit children all over Finland. 

Several clinical drug trials involving rare diseases are currently ongoing at New Children's Hospital. The diseases include skeletal development and growth disorders such as achondroplasia and hypophosphatasia, cardiac and kidney diseases, and pediatric cancers. Many of the trials study treatments targeting the disease mechanism.