Scioderm has started a phase III double-blind, randomized placebo-controlled trial evaluating the 6% concentration, which is being conducted in the United States and Europe and is still recruiting participants. Endpoints are similar to the phase IIb study.
Although not a disease modifying agent, allantoin 6% cream is the biggest treatment advance so far for these patients, Dr. Paller commented. “There is lots of research going on,” including stem cell therapy leading to fibroblast differentiation, grafting cultured keratinocytes onto skin, and intravenous collagen, she said.
But these approaches are in the early stages of research, she added, “and so far we either don’t know their effect or [if] they offer minimal assistance. The concept of using something that has virtually no risk, even if it simply helps make someone more comfortable with less itching and pain, or which starts to enable some healing, is really important to a child with one of these devastating disorders.”
Dr. Paller is also excited about the possibility that starting the treatment earlier, or continuing for a longer time, could even have a better result. While she cautioned that this is a “very small study of patients with mixed EB types,” she said, “still, it’s the best thing we have ever seen with anything topically.”
Dr. Paller is a consultant for Scioderm, which is developing Zorblisa and sponsoring the studies. Northwestern was one of the study sites for the phase IIb study.