CTDH CITES TRIAL DESIGN FOR NIEMANN-PICK TYPE C TREATMENT
CTDH CITES RESPONSES FROM UK MEDICINES REGULATORY AGENCY
CLINICAL TRIAL DESIGN GETS POSITIVE RESPONSES
CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s Medicines and Healthcare Products Regulatory Agency
2015-12-14 12:00:00.158 GMT
CTD Holdings' Clinical Trial Design Receives Positive Responses From U.K.'s
Medicines and Healthcare Products Regulatory Agency
Responses Assist CTD in Its Clinical Trial Application for
Trappsol(R) Cyclo(TM) as a Treatment for Niemann-Pick Type C Disease
ALACHUA, FL -- (Marketwired) -- 12/14/15 -- 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 fa
ilure.
"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.
