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

Mandos

Status and phase: Terminated; Phase 3; Phase 2

Conditions: Niemann-Pick Disease, Type C

Treatments: Drug: Adrabetadex

Study type: Interventional

Funder types: Industry

Identifiers

NCT04958642

VTS301 (Part C)

2015-002548-15 (EudraCT Number)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 66 patients

Sex: All

Ages: 4 to 21 years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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66 participants in 1 patient group

Adrabetadex

Experimental group

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.

Treatment:

Drug: Adrabetadex