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Hand of child with epidermolysis bullosa being examined by a doctor with an reflecting microscope

About clinical trials

Clinical trials are very demanding next to the clinical routine in hospitals. However, they also are drivers in translational research and drug development. Commitment and contribution of medical staff and patients are essential for steady progress in EB treatment.

As of now, there is no causal systemic, long-lasting therapy for EB available. Until such is achieved, new approaches for the symptomatic treatment or local causal therapies will continue to be developed. They aim to increase the patient’s quality of life and correct the underlying gene defect locally in highly affected areas, respectively. In order to translate promising results from the lab to the clinics (“Bench-2-Bedside”), clinical trials are conducted. Depending on the trial phase (phase I-IV), the main focus is on safety assurance, dosage finding, efficacy testing, and long-term tolerance observations (see figure below).

The aim of a clinical trial is market authorization and therefore, the availability of effective therapies for patients. To achieve this high aim a large number of participants are needed. The patient cohort in an orphan disease such as EB is small, hence the demands for multicentric, international trials. Here EB-CLINET can be of help in facilitating communication and connection of clinicians and expertise.

To be able to handle the increased time, administrative and financial effort during clinical trials, a well-trained team of physicians, nurses, and study coordinators are needed. Before the inclusion of patients, competent authorities and an ethics committee must release all study material (study protocol, informed consent forms, investigator medicinal product dossier, questionnaires, and information material). Eligible patients (defined by inclusion and exclusion criteria) must be compliant with the study protocol. Each intervention and examination has to be documented in detail following Good Clinical Practice. After completion, the trial results are evaluated and statistically analyzed and should be published.

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