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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT04958642
Registration number
NCT04958642
Ethics application status
Date submitted
29/06/2021
Date registered
12/07/2021
Titles & IDs
Public title
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
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Scientific title
A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-ß-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
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Secondary ID [1]
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2015-002548-15
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Secondary ID [2]
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VTS301 (Part C)
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Niemann-Pick Disease, Type C
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Condition category
Condition code
Neurological
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Neurodegenerative diseases
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Mental Health
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Other mental health disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Metabolic and Endocrine
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0
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Metabolic disorders
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Neurological
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Other neurological disorders
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Metabolic and Endocrine
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0
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Other metabolic disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Adrabetadex
Experimental: Adrabetadex - All participants receive their prescribed dose of adrabetadex. Dose is allowed to be adjusted down to a minimum of 400 milligrams (mg) or up to a maximum of 900 mg, at the investigator's discretion.
Treatment: Drugs: Adrabetadex
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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Assessment method [1]
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An AE was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
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Timepoint [1]
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Baseline up to 5 years
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Eligibility
Key inclusion criteria
Inclusion criteria:
One of the following is required for inclusion into VTS301 Part C:
* Has agreed to convert from the monthly dosing regimen used in the NIH phase 1/2a protocol to an every 2 weeks dosing regimen
* The investigator has received prior written authorization from the sponsor for the participant to enter VTS301 Part C on an amended dose and/or regimen
* Has received prior written authorization from Vtesse to enroll directly into Part C
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Minimum age
4
Years
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Maximum age
21
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
Exclusion criteria:
* None of the inclusion criteria are applicable
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Study design
Purpose of the study
Treatment
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Allocation to intervention
NA
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Stopped early
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/12/2015
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
11/04/2022
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Sample size
Target
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Accrual to date
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Final
66
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,VictriaWA
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Recruitment hospital [1]
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The Prince of Wales Hospital - Randwick
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Recruitment hospital [2]
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Monash Medical Centre - Clayton
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Recruitment hospital [3]
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Royal Melbourne Hospital - Melbourne
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Recruitment hospital [4]
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Royal Perth Hospital - Perth
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment postcode(s) [2]
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3168 - Clayton
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Recruitment postcode(s) [3]
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- Melbourne
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Recruitment postcode(s) [4]
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6000 - Perth
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Alabama
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United States of America
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California
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Colorado
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District of Columbia
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Florida
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United States of America
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Illinois
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United States of America
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Maryland
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United States of America
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Massachusetts
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New York
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North Carolina
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Texas
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Washington
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France
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Cedex 12
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Germany
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Bochum
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Germany
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Mainz
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Germany
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Münster
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New Zealand
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Hamilton
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Singapore
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Singapore
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Switzerland
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Bern
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Turkey
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Ankara
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United Kingdom
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Birmingham
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Mandos LLC
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844. The trial's final results for the primary outcome measure of Adverse Events (AE) will be reported here. This study is to evaluate how safe and effective adrabetadex is for participants with Niemann-Pick Type C1 (NPC1) disease who experience neurologic symptoms (listed under Keywords). In Parts A/B (NCT02534844), two out of every 3 participants will receive the study drug. The third participant will receive 1 to 2 small needle pricks at the location where the IT injection is normally made (sham control). In Part C, all participants will receive study drug.
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Trial website
https://clinicaltrials.gov/study/NCT04958642
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Study Lead
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Address
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Mandos LLC
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Phone
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Fax
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Email
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Contact person for public queries
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/42/NCT04958642/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/42/NCT04958642/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT04958642