No new therapy without a clinical trial

Clinical trials

At present, no effective therapies exist for any form of EB. Although the current number of EB clinical trials being undertaken is still small, the diversity of therapeutic approaches in preclinical development and entering clinical trials is growing rapidly.

In 2006, there was one pilot clinical trial for a gene therapy in one patient – the first time there was proof that the underlying genetic fault in one type of EB could be corrected. Today, there are tens of clinical trials underway or planned, for all types of EB, using many different approaches to therapy, including cell-, gene-, protein- and drug therapies.

Currently, five phase 3 EB clinical trials are running worldwide. Four of these are sponsor studies and the logos of the associated industry partners can be found below.

21 further trials are active or recruiting patients. As it is not always easy to keep track of the current status of all clinical trials, we cannot guarantee that the diagram below is up-to-date. We have integrated those intervention trials that are currently enrolling patients or, to our knowledge, will start/continue soon. The overview shows how many studies are active in phase I, II and III according to the type of application.

Clinical trials registers

There are several official clinical trial websites providing information on EB clinical trials worldwide. These include information about location, clinical lead, and trial sponsor, as well as the trial purpose, how the outcomes will be measured, and inclusion/exclusion criteria. 

Links to registers

• US-based ClinicalTrials.gov: clinicaltrials.gov

• EU Clinical Trials Register (EUDRACT): clinicaltrialsregister.eu

• International Clinical Trials Registry Platform (ICTRP):  apps.who.int/trialsearch