Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Launched by CYCLO THERAPEUTICS, INC. · Mar 26, 2019

Keywords

ClinConnect Summary

This is an open-label extension study of intravenous HP-β-CD (administered as Trappsol® Cyclo(TM)) in patients with NPC-1 to evaluate long-term safety and efficacy by providing continued access to treatment following completion of study CTD-TCNPC-101. Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator. Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol.

Thereafter, vi...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Completion of study CTD-TCNPC-101 with no safety concerns at the final visit
• • Negative urine pregnancy test for females of child bearing potential
• • Patients must be legally resident in the USA with access to healthcare
• • Written, informed consent
• Exclusion Criteria:
• • Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent)
• • Concurrent medical conditions representing a contraindication to any of the study medications
• • Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) \< 60mL/min/1.73m2
• • Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) \>1.8
• • Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up visit