Investigating the role of Miglustat in the management of a patient with Tangier’s Disease: a single case experiment.
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.
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.
This is an n-of-1 study of ABAB design, with alternating periods of intervention and control.
In progress, closed to recruitment.
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)
Senior Trial Manager:
Frances Webley
Trial Coordinator:
Tom Oliver
Email: [email protected]
Email: [email protected]
Esstential Trial Documents
MUSTANG Protocol v3 03-Mar-2022
MUSTANG GP Letter v1 11-Dec-2020
MUSTANG Consent Form v3 03-Mar-2022
MUSTANG Dietary Diary v1 21-Jan 2021
MUSTANG Overall Neuropathy Limitations Scale v2 05-Mar-2021
MUSTANG EQ-5D-5L Paper Self-Complete v1.2
MUSTANG EQ-5D-5L Paper Interviewer Administration v1.4
MUSTANG Pregnant Partner PIS v0.3 12-Aug-2021
MUSTANG Pregnant Partner consent form v0.1 19-May-2021
ISF Documents
MUSTANG -Instructions for Adverse and Serious Adverse Event Reporting v1 09-Mar-2021
MUSTANG - SAE-SUSAR Report Form v1 09-Mar-2021
MUSTANG - Adverse Event Form v1 11-Mar-2021
MUSTANG - Master Patient List v1 26-Nov-2020
MUSTANG - Pre-Visit Checklists v1 16-Feb-2021
MUSTANG - Site Visit Log v1 04-Jan-2021
MUSTANG - Study Contacts and Information Page v1 27-Nov-2020
IMP Documents
MUSTANG - Pharmacy Manual v1 20-May-2021
MUSTANG - Drug Accountability Log v2 01-Apr-2021
MUSTANG - Summary of Product Characteristics (SmPC) 18-Nov 202
Laboratory Documents
MUSTANG - Laboratory Manual v3 20-Apr-2021
MUSTANG - Plasma Sample Storage Log v1 24-Feb-2021
MUSTANG - Urine Sample Storage Log v1 12-Mar-2021
MUSTANG - Sample Shipment Log - Dried Blood Sport Filtercard v1 25-Feb-2021
MUSTANG - Sample Shipment Log - EDTA 10ml v1 04-Mar-2021
MUSTANG - Sampling Worksheet - EDTA 10ml for Platt Lab
MUSTANG - Sampling Worksheet - Li-heparin (6ml), Random Urine (5ml) for GOH
MUSTANG - Willink Referral Form 1
Case Report Forms
MUSTANG - Trial Registration Form v1 09-Feb-2021
MUSTANG - End of Study CRF v2 31-Mar-2021
MUSTANG - Pregnancy Report Form v1 31-Mar-2021
MUSTANG - CRF 01 Eligibility, Registration and Baseline v2 31-Mar-2021
MUSTANG - CRF 02 Patient Contact Details v2 31-Mar-2021
MUSTANG - CRF 03 Concomitant Medications v2 31-Mar-2021
MUSTANG - CRF 04 Medical History v2 31-Mar-2021
MUSTANG - CRF 05 Month 2, v2 31-Mar-2021
MUSTANG - CRF 06 Month 4, v2 31-Mar-2021
MUSTANG - CRF 07 Month 6, v2 31-Mar-2021
MUSTANG - CRF 08 Month 8, v2 31-Mar-2021
MUSTANG - CRF 09 Month 10, v2 31-Mar-2021
MUSTANG - CRF 10 Month 12, v2 31-Mar-2021
MUSTANG - CRF 11 Month 14, v2 31-Mar-2021
MUSTANG - CRF 12 Month 16, v2 31-Mar-2021
MUSTANG - CRF 13 Month 18, v2 31-Mar-2021
MUSTANG - CRF 14 Month 20, v2 31-Mar-2021
MUSTANG - CRF 15 Month 22, v2 31-Mar-2021
MUSTANG - CRF 16 Month 24, v2 31-Mar-2021