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

NCT03893071 | Unknown Phase 1 FDA Drug

• 1 other identifier: CTD-TCNPC-102
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Niemann-Pick Disease, Type C1

Outcome Measures

Primary Outcomes (4)

• Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product

  Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product

  1-104 weeks

• Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product

  Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product

  1-104 weeks

• Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation

  Discontinuation, Study and Site Discontinuation

  1-104 weeks

• Auditory capacity will be measured by behavioral auditory assessment

  Auditory capacity will be measured by behavioral auditory assessment

  1-104 weeks

Secondary Outcomes (1)

• Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)

  1-104 weeks

Study Arms (1)

Hydroxypropyl-β-cyclodextrin IV

EXPERIMENTAL

Hydroxypropyl-β-cyclodextrin will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.

Drug: Hydroxypropyl-β-cyclodextrin

Interventions

Hydroxypropyl-β-cyclodextrin DRUG

HP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.

Also known as: Hydroxypropyl-β-cyclodextrin (HP-β-CD), Trappsol® Cyclo

Hydroxypropyl-β-cyclodextrin IV

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Sponsors & Collaborators

• Cyclo Therapeutics, Inc.: lead

Study Sites (1)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Related Publications (1)

• Hastings C, Liu B, Hurst B, Cox GF, Hrynkow S. Intravenous 2-hydroxypropyl-beta-cyclodextrin (Trappsol(R) Cyclo) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. Mol Genet Metab. 2022 Dec;137(4):309-319. doi: 10.1016/j.ymgme.2022.10.004. Epub 2022 Oct 17.

  PMID: 36279795 DERIVED

MeSH Terms

Conditions

Niemann-Pick Disease, Type C

Condition Hierarchy (Ancestors)

Niemann-Pick Diseases; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• Caroline Hastings, MD

  UCSF Benioff Children's Hospital Oakland

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2019
• First Posted: March 28, 2019
• Study Start: May 23, 2019
• Primary Completion: March 1, 2022
• Study Completion: March 1, 2022
• Last Updated: June 18, 2021

Record last verified: 2021-02

Locations

US (1)