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

Niemann-Pick Disease, Type C Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04958642
• Other study ID #: VTS301 (Part C)
• Secondary ID: 2015-002548-15
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: December 23, 2015
• Est. completion date: April 11, 2022

Study information

• Verified date: August 2023
• Source: Mandos LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 66
• Est. completion date: April 11, 2022
• Est. primary completion date: April 11, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years to 21 Years

Eligibility

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

Related Conditions & MeSH terms

• Niemann-Pick Disease, Type C
• Niemann-Pick Diseases
• Pick Disease of the Brain

Intervention

Drug:
• Adrabetadex
Mallinckrodt test formulation, administered intrathecal (IT) via lumbar puncture (LP) infusion.

Locations

Country	Name	City	State
Australia	Monash Medical Centre	Clayton	Victoria
Australia	Royal Melbourne Hospital	Melbourne	Victria
Australia	Royal Perth Hospital	Perth	Western Australia
Australia	The Prince of Wales Hospital	Randwick	New South Wales
France	CHU Paris Est - Hopital d'Enfants Armand-Trousseau	Paris	Cedex 12
Germany	Katholisches Klinikim Bochum gGmbH	Bochum
Germany	Universitaetsklinikum Mainz	Mainz
Germany	Universitaetsklinikum Muenster	Münster
New Zealand	Waikato Hospital	Hamilton
Singapore	National University Hospital (Singapore) Pte, Ltd	Singapore
Switzerland	INSELSPITAL, Universitätsspital Bern	Bern
Turkey	Gazi University Medical Faculty	Ankara
Turkey	Hacettepe University Medical Faculty	Ankara
United Kingdom	Birmingham Children's Hospital NHS Foundation Trust	Birmingham
United Kingdom	Great Ormond Street Hospital	London
United States	Lehigh Valley Health Network	Allentown	Pennsylvania
United States	University of Colorado Denver	Aurora	Colorado
United States	Dell Children's Medical Center of Central Texas	Austin	Texas
United States	Eunice Kennedy Shriver National Institute of Child Health and Human Development	Bethesda	Maryland
United States	University of Alabama at Birmingham	Birmingham	Alabama
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Montefiore Medical Center	Bronx	New York
United States	University of North Carolina	Chapel Hill	North Carolina
United States	Rush University Medical Center	Chicago	Illinois
United States	Shands Children's Hospital	Gainesville	Florida
United States	Children's Hospital of Orange County: CHOC Children's	Orange	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of California San Francisco	San Francisco	California
United States	Multicare Institute for Research and Innovation	Tacoma	Washington
United States	Children's National Medical Center	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Mandos LLC

Countries where clinical trial is conducted

United States,  Australia,  France,  Germany,  New Zealand,  Singapore,  Switzerland,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Baseline up to 5 years