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

Phase 3TerminatedNCT04958642

Mandos LLC66 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Niemann-Pick Disease, Type C

Interventions

Eligibility

Sex: ALLMin age: 4 YearsMax age: 21 Years

Medical Language ↔ Plain English

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

Locations (30)

University of Alabama at Birmingham

Birmingham, Alabama, United States

Children's Hospital of Orange County: CHOC Children's

Orange, California, United States

University of California San Francisco

San Francisco, California, United States

University of Colorado Denver

Aurora, Colorado, United States

Children's National Medical Center

Washington D.C., District of Columbia, United States

Outcomes

Primary Outcomes

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.

Time frame: Baseline up to 5 years