Fistulas are sores between the end of the bowel and the skin near the anus. Cx601 uses stem cells to treat them after other therapies have failed. Fistulas can cause bleeding and discharge when defecating, and can be painful.
Previous clinical trials, including the Phase 3 ADMIRE-CD (NCT01541579) study, showed that Cx601 was able to eliminate fistulas that other therapies could not. The trials also showed Cx601 to be safe.
The new global study is designed to confirm the effectiveness and safety of a single administration of Cx601 as a treatment for fistulas.
TiGenix hopes the results are good enough for it to submit a Biologic License Application for Cx601 to the U.S. Food and Drug Administration.
The company announced the new trial after convening its first meeting of European clinical trial investigators in Rome, June 8 and 9. The meeting brought together more than 60 gastroenterologists, colorectal surgeons and study coordinators from 30 trial sites across the European Union. TiGenix plans similar meetings in other European countries, the United States, Canada and Israel, starting later this year.
“Investigator meetings are critical to the success of any clinical trial,” Marie-Paule Richard, TiGenix’s chief medical officer, said in a press release. “The attendance and support from leading experts in the GI [gastro-intestinal] field confirms the strong interest from the medical community in our global phase III trial and, ultimately, for Cx601 as a potential new treatment for patients.”
After being principal investigator of the “European ADMIRE-CD trial, which successfully met its primary endpoint and safety and efficacy profile, I am excited to participate in this global trial as study coordinator for Europe and Israel,” said Dr. Julian Panés, head of gastroenterology at Hospital Clínic de Barcelona. “We are eager to continue building on the evidence gained from the ADMIRE-CD trial and, ultimately, to offer patients a durable cure for their complex perianal fistulas, which remain a severe and debilitating complication of Crohn’s disease.”
Results from ADMIRE-CD were published in The Lancet in July 2016 under the title “Expanded allogeneic adipose-derived mesenchymal stem cells (Cx601) for complex perianal fistulas in Crohn’s disease: a phase 3 randomised, double-blind controlled trial.”
TiGenix used the results to submit a marketing authorization application to the European Medicines Agency. The agency has yet to make a decision on the application.