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

• Status: Active, not recruiting
• Trial end date: 2022-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: CTD Holdings, Inc.; Cyclo Therapeutics, Inc.
• Treatments: Emodepside

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