Investigating the role of miglustat in the management of a patient with Tangier Disease

Not Applicable

• Conditions: Tangier disease; Nutritional, Metabolic, Endocrine

• Registration Number: ISRCTN17945917
• Lead Sponsor: niversity Hospitals Birmingham NHS Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-07
• Last Update Posted: 12 July 2021
• Study Completion: 2023-04-28

Recruitment & Eligibility

• Status: Ongoing
• Sex: Male
• Target Recruitment: 1

Inclusion Criteria

This is an n-of-1 study, where the potential participant has already been identified, and is known to the investigators there are limited inclusion and exclusion criteria:
1. Diagnosis of Tangier Disease
2. Aged =18 years
3. Willing and able to provide informed consent.
4. Agrees to use effective contraception throughout the study to avoid fathering a child

Exclusion Criteria

Severe renal impairment

Study & Design

• Study Type: Interventional
• Study Design: Not specified