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

Status unconfirmed · Phase 1

Conditions studied: Niemann-Pick Disease, Type C1

In brief

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.

Key facts

Study ID

NCT03893071

Run by

Cyclo Therapeutics, Inc.

People needed

12

Starts

2019-05-23

Expected to finish

2022-03-01

Last updated by the study team

2021-06-18

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• UCSF Benioff Children's Hospital Oakland — Oakland, California, United States

Full record on ClinicalTrials.gov

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