Clinical trial NCT04958642: Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Official title: A Phase 2B/3 Prospective, Randomized, Double-Blind, Sham-Controlled Trial of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in Subjects With Neurologic Manifestations of Niemann-Pick Type C1 (NPC1) Disease
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Niemann-Pick Disease, Type C
Keywords: Niemann-Pick Type C1 (NPC1) Disease
neurologic disease
gross motor dysfunction
fine motor dysfunction
dysphagia
swallowing problems
cognitive dysfunction
gait abnormalities
pediatrics
Lead sponsor: Mandos LLC
Sponsor class: INDUSTRY
First posted: 2021-07-12
Enrollment: 66
Sex: ALL
Minimum age (years): 4
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: In Part C, all participants receive adrabetadex
Primary purpose: TREATMENT

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.

Detailed description: Participants in Part C will receive adrabetadex until the investigator considers adrabetadex to no longer be beneficial to the participant, or the development program is discontinued.

Eligibility 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

Exclusion criteria:

* None of the inclusion criteria are applicable

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline up to 5 years
Secondary outcomes:
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Orange
country: United States
facility: Children's Hospital of Orange County: CHOC Children's
state: California
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado Denver
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: Shands Children's Hospital
state: Florida
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Bethesda
country: United States
facility: Eunice Kennedy Shriver National Institute of Child Health and Human Development
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Allentown
country: United States
facility: Lehigh Valley Health Network
state: Pennsylvania
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Austin
country: United States
facility: Dell Children's Medical Center of Central Texas
state: Texas
city: Tacoma
country: United States
facility: Multicare Institute for Research and Innovation
state: Washington
city: Randwick
country: Australia
facility: The Prince of Wales Hospital
state: New South Wales
city: Clayton
country: Australia
facility: Monash Medical Centre
state: Victoria
city: Melbourne
country: Australia
facility: Royal Melbourne Hospital
state: Victria
city: Perth
country: Australia
facility: Royal Perth Hospital
state: Western Australia
city: Paris
country: France
facility: CHU Paris Est - Hopital d'Enfants Armand-Trousseau
state: Cedex 12
city: Bochum
country: Germany
facility: Katholisches Klinikim Bochum gGmbH
city: Mainz
country: Germany
facility: Universitaetsklinikum Mainz
city: Münster
country: Germany
facility: Universitaetsklinikum Muenster
city: Hamilton
country: New Zealand
facility: Waikato Hospital
city: Singapore
country: Singapore
facility: National University Hospital (Singapore) Pte, Ltd
city: Bern
country: Switzerland
facility: INSELSPITAL, Universitätsspital Bern
city: Ankara
country: Turkey
facility: Gazi University Medical Faculty
city: Ankara
country: Turkey
facility: Hacettepe University Medical Faculty
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital
Study officials:
affiliation: Mandos LLC
name: Clinical Study Lead
role: STUDY_DIRECTOR
References: