International Niemann–Pick Disease Alliance

CTD Holdings Initiates Recruitment for U.S. Phase I Clinical Study of Trappsol® Cyclo™ for Treatment of Niemann-Pick Disease Type C Patients to be Enrolled at Children’s Hospital & Research Center Oakland

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ALACHUA, FL – (Marketwired) – March 23, 2017 – CTD Holdings, Inc. (OTCQB: CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it has begun recruiting patients at Children’s Hospital & Research Center Oakland for the Company’s Phase I clinical study in the U.S. that will evaluate the intravenous administration of Trappsol® Cyclo™ in patients with Niemann-Pick Disease Type C (NPC), a rare and fatal genetic disease that impacts the brain, lung, liver, spleen, and other organs. Dr. Caroline Hastings, Pediatric Hematologist Oncologist at UCSF Benioff Children’s Hospital Oakland, is the Principal Investigator for the U.S. study. The U.S. clinical study will require 12 patients to be fully enrolled.

To date, intravenous Trappsol® Cyclo™ has been administered to 21 NPC patients worldwide, some for more than six years, via compassionate use programs. Data from treating physicians have demonstrated that multiple patients have shown marked improvements in neurological symptoms, lung function or liver morphology, or had stabilization of disease progression, with no significant safety concerns. Dr. Hastings was the first physician in the U.S. to administer Trappsol® Cyclo™ through compassionate use, beginning in 2009. She shares her original treatment protocol with physicians world-wide.
“As there is no approved treatment for NPC in the U.S., and having worked with so many families over many years using hydroxypropyl beta cyclodextrins compassionately in this patient population, I am pleased to be leading this clinical trial,” said Dr. Hastings, “This formal trial, which will administer Trappsol® Cyclo™ intravenously should provide crucial data that may help further develop this drug and gain market approval, which is so sorely needed in this underserved patient population.”
“The initiation of enrollment in our U.S. clinical site is another significant milestone for the Company in the development of this important treatment for a devastating disease,” said CTD Chairman and CEO, N. Scott Fine. “The opening of the Oakland site complements the recent opening of the Salford, UK site, both of which are positive developments for our clinical program and for the NPC community. We are grateful for the support and encouragement from the many patient families, researchers and clinicians that have worked with us to get to this point.”
Trappsol® Cyclo™ is a parenteral grade of hydroxypropyl beta cyclodextrin, a donut-shaped molecule comprised of seven glucopyranose units. Its hydrophilic exterior allows it to move easily through the body, and its inner hydrophobic cavity allows it to capture and hold certain types of molecules, including cholesterol. In NPC patients, cholesterol accumulates abnormally in the body cells. “NPC is a systemic disease, which is why we are administering Trappsol® Cyclo™ intravenously in this study,” said Dr. Sharon Hrynkow, CTD’s Senior Vice President for Medical Affairs. “We look forward to working with our colleagues in Oakland on this phase I study, which complements the work of colleagues in the EU study in the UK, Sweden and other sites.” The Company previously received Orphan Drug designation for the use of Trappsol® Cyclo™ in NPC from the U.S. Food and Drug Administration and the European Medicines Agency. For further information on this trial or other CTD trials please visit:, or send email to: Dr. Sharon Hrynkow:
For patients interested in learning more about the Oakland-based trial, please email: Dr. Caroline Hastings:, or Cyrus Bascon, Study Coordinator:

For press inquiries contact: Hans Vitzthum: About CTD Holdings: CTD Holdings, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company’s Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease. Additional indications for the active ingredient in Trappsol® Cyclo™, are in development. For additional information, visit the company’s website: Safe Harbor Statement: This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company’s future performance include the company’s ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company’s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.
Hans Vitzthum
LifeSci Advisors, LLC
Source: CTD Holdings, Inc.

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