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

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

Status

Active, not recruiting

Phases

Phase 4

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON37985

Enrollment

Registered

2011-07-20

Start date

2011-08-03

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neutral lipid storage disease with myopathy / heriditary fataccumulation disorder

Interventions

Patients will receive a dosage of 400mg Bezafibrate every day during 28 weeks. Control subjects will not be treated.

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

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

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.

Design outcomes

Primary

Measure	Time frame
Main study parameters are the changes in mitochondrial function, cardiac, hepatic and muscle lipid accumulation and cardiac function after treatment.	—

Secondary

Measure	Time frame
As secondary endpoints changes in insulin sensitivity is considered.	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)