Clinical trials are a crucial part of finding treatments and cures for ectodermal dysplasias. Clinical research is the way that drugs, devices or other treatments are tested in humans to see if they are safe and effective.  A clinical trial may find that a new strategy, treatment, or device improves patient outcomes, offers no benefit or
causes unexpected harm.

Edimer Pharmaceuticals’ XLHED Newborn Clinical Trial began in 2013 and was the first one to test a potential treatment for an ectodermal dysplasia. They tested the use of EDI200, a protein therapy, to treat the symptoms of 10 newborns affected by x-linked hypohidrotic ectodermal dysplasia (XLHED). That trial completed recruitment in 2015. They didn’t achieve the expected outcomes but continue to collect long-term data. There were some modest signs of improvement in biologic activity, meaning the development and function of various body parts that XLHED affects. However, they didn’t see significant changes in sweat gland function and other early markers of biologic activity. Edimer and the research investigators involved hypothesize that the babies didn’t receive the protein early enough to have a significant impact. Edimer continues to have ongoing discussions with the investigators to find a way to explore earlier dosing. Dr. Holm Schneider dosed twin boys affected by XLHED in utero in the spring of 2016. We await the results to see if this treatment is effective.

Recruitment

Recruitment for clinical trials can often take a long period of time, especially for rare disorders like ectodermal dysplasias. Our Ectodermal Dysplasias International Registry can speed up that process. It was a key first step not for the XLHED Newborn Clinical Trial, and is for all future ectodermal dysplasias clinical studies. That is why it’s important that every person affected by an ectodermal dysplasia join the Registry and share their information.

We will share information about any clinical trials for ectodermal dysplasias. A key component to these trials will be for affected individuals to be willing to participate in research.

Learn more about clinical trials at ClinicalTrial.gov.