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Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

Primary Purpose

Niemann-Pick Disease, Type C

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Adrabetadex
Sponsored by
Mandos LLC
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Niemann-Pick Disease, Type C focused on measuring Niemann-Pick Type C1 (NPC1) Disease, neurologic disease, gross motor dysfunction, fine motor dysfunction, dysphagia, swallowing problems, cognitive dysfunction, gait abnormalities, pediatrics

Eligibility Criteria

4 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Alabama at Birmingham
  • Children's Hospital of Orange County: CHOC Children's
  • University of California San Francisco
  • University of Colorado Denver
  • Children's National Medical Center
  • Shands Children's Hospital
  • Rush University Medical Center
  • Eunice Kennedy Shriver National Institute of Child Health and Human Development
  • Boston Children's Hospital
  • Montefiore Medical Center
  • University of North Carolina
  • Lehigh Valley Health Network
  • The Children's Hospital of Philadelphia
  • Dell Children's Medical Center of Central Texas
  • Multicare Institute for Research and Innovation
  • The Prince of Wales Hospital
  • Monash Medical Centre
  • Royal Melbourne Hospital
  • Royal Perth Hospital
  • CHU Paris Est - Hopital d'Enfants Armand-Trousseau
  • Katholisches Klinikim Bochum gGmbH
  • Universitaetsklinikum Mainz
  • Universitaetsklinikum Muenster
  • Waikato Hospital
  • National University Hospital (Singapore) Pte, Ltd
  • INSELSPITAL, Universitätsspital Bern
  • Gazi University Medical Faculty
  • Hacettepe University Medical Faculty
  • Birmingham Children's Hospital NHS Foundation Trust
  • Great Ormond Street Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Adrabetadex

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
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.

Secondary Outcome Measures

Full Information

First Posted
June 29, 2021
Last Updated
August 4, 2023
Sponsor
Mandos LLC
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1. Study Identification

Unique Protocol Identification Number
NCT04958642
Brief Title
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

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Terminated
Why Stopped
Terminated by previous Sponsor decision
Study Start Date
December 23, 2015 (Actual)
Primary Completion Date
April 11, 2022 (Actual)
Study Completion Date
April 11, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mandos LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Model Description
In Part C, all participants receive adrabetadex
Masking
None (Open Label)
Allocation
N/A
Enrollment
66 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Adrabetadex
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Adrabetadex
Other Intervention Name(s)
VTS-270, 2-hydroxypropyl-β-cyclodextrin, Cyclodextrin
Intervention Description
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.
Primary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Description
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.
Time Frame
Baseline up to 5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Study Lead
Organizational Affiliation
Mandos LLC
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama at Birmingham
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Children's Hospital of Orange County: CHOC Children's
City
Orange
State/Province
California
ZIP/Postal Code
92867
Country
United States
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143-0780
Country
United States
Facility Name
University of Colorado Denver
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Children's National Medical Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Shands Children's Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Rush University Medical Center
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Eunice Kennedy Shriver National Institute of Child Health and Human Development
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892-2425
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
University of North Carolina
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27514
Country
United States
Facility Name
Lehigh Valley Health Network
City
Allentown
State/Province
Pennsylvania
ZIP/Postal Code
18101
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Dell Children's Medical Center of Central Texas
City
Austin
State/Province
Texas
ZIP/Postal Code
78723
Country
United States
Facility Name
Multicare Institute for Research and Innovation
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Facility Name
The Prince of Wales Hospital
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Facility Name
Monash Medical Centre
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Melbourne
State/Province
Victria
Country
Australia
Facility Name
Royal Perth Hospital
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Facility Name
CHU Paris Est - Hopital d'Enfants Armand-Trousseau
City
Paris
State/Province
Cedex 12
ZIP/Postal Code
75 571
Country
France
Facility Name
Katholisches Klinikim Bochum gGmbH
City
Bochum
ZIP/Postal Code
44791
Country
Germany
Facility Name
Universitaetsklinikum Mainz
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Universitaetsklinikum Muenster
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Waikato Hospital
City
Hamilton
ZIP/Postal Code
3204
Country
New Zealand
Facility Name
National University Hospital (Singapore) Pte, Ltd
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Facility Name
INSELSPITAL, Universitätsspital Bern
City
Bern
ZIP/Postal Code
3010
Country
Switzerland
Facility Name
Gazi University Medical Faculty
City
Ankara
Country
Turkey
Facility Name
Hacettepe University Medical Faculty
City
Ankara
Country
Turkey
Facility Name
Birmingham Children's Hospital NHS Foundation Trust
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Great Ormond Street Hospital
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease

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