MUSTANG

Title

Investigating the role of Miglustat in the management of a patient with Tangier’s Disease: a single case experiment.

Description

MUSTANG is an n-of-1 trial of ABAB design which aims to determine, in a single patient, the efficacy of Miglustat to improve cholesterol metabolism and resulting neuropathy caused by Tangier disease.

Objectives

Primary Objective

·       To determine the efficacy of Miglustat to improve arm fine motor control, finger dexterity and grip strength. 

Secondary Objective

·       To determine the efficacy of Miglustat to improve Quality of Life, activities of daily living, aerobic capacity, endurance, ambulation and nerve conduction.

Mechanistic Objective

·       To investigate the potential mechanisms by which Miglustat does, or does not, have an effect.

Trial Design

This is an n-of-1 study of ABAB design, with alternating periods of intervention and control.

Trial Status

In progress, closed to recruitment.

Population

A single patient with Tangier disease, who is already known to the chief investigator, and keen to try the intervention.

This trial is funded by a National Institute for Health and Care Research grant (award reference no. NIHR132844)