In the field of endocrinology, phase 2 and 3 clinical studies are conducted with a focus on rare endocrine and metabolic diseases. Based on years of specialized care of children and adolescents from all over Germany, patients and their families are informed directly about the possibility of participation.

An experienced qualified (GCP, MPG certificates) team is available for selected rare diseases both in the medical and nursing area as well as in the study coordination and documentation.

Special experience is available in congenital hyperinsulinism: the interdisciplinary center COACH is one of six certified centers of excellence worldwide in which innovative therapeutic approaches are being tested in registration studies. Effective drugs, especially for children with diazoxide-resistant diffuse forms of congenital hyperinsulinism, are still lacking. At our center, various therapeutic approaches are being investigated in phase 2 and 3 trials: Insulin receptor antibodies, stabilized glucagon, and others: (ClinicalTrials.gov: NCT04538989; NCT04732416; NCT04172441; NCT03941236).

Furthermore, clinical trials are being conducted in achondroplasia and other congenital skeletal system disorders (NCT03424018; NCT03197766; NCT03794609). In September 2021, based on the studies also conducted at our center, the European Medicines Agency (EMA) approved for the first time, a drug for the treatment of this distinct form of dwarfism for children aged 2 years and older (Voxzogo).

As a member of the European Reference Networks (ERN) Endo-ERN, Metab-ERN und ERN-BOND the Endocrinology/Diabetology Unit is a pediatric center for registry studies: https://eurreca.net/.