The effect of fibrate therapy in two patients with neutral lipid storage disease with myopathy (NLSDM).

Phase 4

Completed

• Conditions: Neutral lipid storage disease with myopathy / heriditary fataccumulation disorder; 10019280; 10027424; 10013317

• Registration Number: NL-OMON37985
• Lead Sponsor: niversiteit Maastricht

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 29 April 2024

Recruitment & Eligibility

• Status: Completed
• Sex: Not specified
• Target Recruitment: 10

Inclusion Criteria

NLSDM:
- Patients with NLSDM;Controls:
- female gender
- between 20-45 years old
- BMI between 20-30 kg/m2
- healthy (no chronic diseases)

Exclusion Criteria

NLSDM:
- none;Controls:
- use of Fibrates and anti-coagulantia
- Contra-indications for MRI:
- Electronic implants such as pacemakers or neurostimulator
- Iron-containing corpora aliena in eyes or brain
- Some hearing aids and artificial (heart) valves which are contraindicated for MRS
- Claustrophobia
- Subjects, who do not want to be informed about unexpected medical findings, or do not wish that their physician is informed, cannot participate in the study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Main study parameters are the changes in mitochondrial function, cardiac,<br /><br>hepatic and muscle lipid accumulation and cardiac function after treatment. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>As secondary endpoints changes in insulin sensitivity is considered.</p><br>