CTD Holdings, Inc. (OTCQB: CTDH), a biotechnology company that develops cyclodextrin-based products for the treatment of disease, has received positive responses from a major U.K. healthcare regulator to the clinical trial design for its Trappsol® Cyclo™ drug, which is used to treat Niemann-Pick Type C (NPC). NPC is a rare and fatal cholesterol metabolism disease found primarily in children and young adults.
The Medicines and Healthcare Products Regulatory Agency (MHRA) provided positive responses to CTD's major clinical study design elements, including safety and toxicity, patient inclusion and exclusion criteria, and biochemical analysis and clinical outcomes. The MHRA advice holds that CTD has sufficient pre-clinical data to support its clinical trial. The MHRA's responses follow its Scientific Advice Meeting with CTD in November.
CTD will prepare a formal clinical trial application for the MHRA at which time CTD may receive additional feedback. CTD will select sites in the U.K. for its Phase II trial and expects to begin enrolling U.K. patients in early 2016. The company also plans to enroll patients in other European countries and the U.S. at a later date.
"MHRA's responses are a powerful signal to NPC patients, families and CTD stakeholders that our Phase II clinical study design is on target," said CTD Chairman and CEO N. Scott Fine. "We are pleased with the positive reception and look forward to submitting our clinical trial application to the MHRA."
CTD's clinical study design calls for intravenous administration of Trappsol® Cyclo™ (hydroxypropyl beta cyclodextrin) in NPC patients two years of age and older. The study will examine the effect of Trappsol® Cyclo™ on biochemical markers of cholesterol metabolism and clearance post-administration, as well as the drug's effect on neurologic, lung and liver symptoms. NPC disease causes the build-up of cholesterol in every cell of the body, leading to cognitive and movement disorders, liver dysfunction and major organ failure.
"The elements of CTD's clinical study design were drawn largely from protocols using Trappsol® Cyclo™ in compassionate use programs around the world," said Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs. "We are particularly grateful to the NPC families and physicians from the U.S., Brazil and Spain who shared their data and experiences with us, including those who have been administering Trappsol® Cyclo™ intravenously for more than five years."
CTD plans to engage with additional regulatory agencies in early 2016.