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

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ClinicalTrials.gov Identifier: NCT03893071
Recruitment Status : Recruiting
First Posted : March 28, 2019
Last Update Posted : March 28, 2019
Sponsor:
Information provided by (Responsible Party):
CTD Holdings, Inc.

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.

Condition or disease Intervention/treatment Phase
Niemann-Pick Disease, Type C1 Drug: Hydroxypropyl-β-cyclodextrin Phase 1

Detailed Description:

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, visits to the parent site will be every 6 months until cessation. The study will continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient discontinuation. All patients who complete study CTD‐TCNCP‐101 and pass the screening criteria will be eligible for this study. The study will be directed from parent site in the US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.

For logistical reasons home infusions will be permitted as long as AEs, infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines. A Safety Review Committee (SRC) consisting of an independent advisor, the medical monitor, and the principal investigator will be established to review AEs and laboratory data throughout the study.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open‐Label Extension Study of the Long‐Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP‐β‐CD) in Patients With Niemann‐Pick Disease Type C (NPC‐1)
Estimated Study Start Date : March 2019
Estimated Primary Completion Date : March 2021
Estimated Study Completion Date : March 2021


Arm Intervention/treatment
Experimental: Hydroxypropyl-β-cyclodextrin IV
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
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.
Other Names:
  • Hydroxypropyl-β-cyclodextrin (HP-β-CD)
  • Trappsol® Cyclo




Primary Outcome Measures :
  1. Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product [ Time Frame: 1-104 weeks ]
    Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product

  2. Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product [ Time Frame: 1-104 weeks ]
    Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product

  3. Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation [ Time Frame: 1-104 weeks ]
    Discontinuation, Study and Site Discontinuation

  4. Auditory capacity will be measured by behavioral auditory assessment [ Time Frame: 1-104 weeks ]
    Auditory capacity will be measured by behavioral auditory assessment


Secondary Outcome Measures :
  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) [ Time Frame: 1-104 weeks ]
    Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03893071


Contacts
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Contact: Sharon H Hrynkow, PhD 3869657273 sharon.hrynkow@cyclodex.com
Contact: Karen Rabb +12144763754 Karen.Rabb@synteract.com

Locations
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United States, California
UCSF Benioff Children's Hospital Oakland Recruiting
Oakland, California, United States, 94609
Contact: Dr Caroline Hastings         
Sponsors and Collaborators
CTD Holdings, Inc.
Investigators
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Principal Investigator: Caroline Hastings, MD UCSF Benioff Children's Hospital Oakland

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Responsible Party: CTD Holdings, Inc.
ClinicalTrials.gov Identifier: NCT03893071     History of Changes
Other Study ID Numbers: CTD‐TCNPC‐102
First Posted: March 28, 2019    Key Record Dates
Last Update Posted: March 28, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Frontotemporal Lobar Degeneration
Dementia
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations
Signs and Symptoms
TDP-43 Proteinopathies
Neurodegenerative Diseases
Proteostasis Deficiencies
Metabolic Diseases
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Histiocytosis, Non-Langerhans-Cell
Histiocytosis