The effect of lipid lowering medication on lipid accumulation in patients with neutral lipid storage disease with muscle weakness.

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2011-001205-27-NL
• Date of registration: 25/07/2011
• Prospective Registration: Yes
• Public title: The effect of lipid lowering medication on lipid accumulation in patients with neutral lipid storage disease with muscle weakness.
• Scientific title: The effect of fibrate therapy in two patients with neutral lipid storage disease with myopathy (NLSDM). - Fibrate Trail
• Date of first enrolment: 04/08/2011
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-001205-27
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: yes Other trial design description: case study (number of patients is 2) If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Netherlands

Key inclusion & exclusion criteria

Inclusion criteria:
Patients need to have NLSDM; as there are only 6 patients in the world and 2 of them in the Netherlands, we aim to include both patients.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
This is more a case study, therefore we don't have any exclusion criteria.


Age minimum:
Age maximum:
Gender:
Female: no
Male: no

Health Condition(s) or Problem(s) studied

Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Neutral lipid storage disease with myopathy
MedDRA version: 13.1 Level: PT Classification code 10007636 Term: Cardiomyopathy System Organ Class: 10007541 - Cardiac disorders
MedDRA version: 13.1 Level: HLGT Classification code 10013317 Term: Lipid metabolism disorders System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 13.1 Level: PT Classification code 10028641 Term: Myopathy System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders

Intervention(s)

Trade Name: Bezalip Retard
Product Name: Bezalip Retard
Product Code: RVG 18388
Pharmaceutical Form: Tablet
INN or Proposed INN: BEZAFIBRATE
CAS Number: 41859-67-0
Current Sponsor code: Bezalip
Other descriptive name: none
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 400-

Primary Outcome(s)

Secondary Objective: Secondary objectives are to improve hepatic and skeletal muscle insulin sensitivity,
thereby reducing morbidities and mortality in NLSDM patients.

Primary end point(s): Main study parameters are the changes in mitochondrial function, cardiac, hepatic and
muscle lipid accumulation and cardiac function after treatment.

Timepoint(s) of evaluation of this end point: These endpoints will be evaluated after 12 weeks.

Main Objective: We would like to evaluate the beneficial effects of fibrate treatment on muscle mitochondrial and cardiac function in patients with NLSDM.

Secondary Outcome(s)

Secondary end point(s): As secondary endpoints changes in insulin sensitivity is considered.

Timepoint(s) of evaluation of this end point: also after 12 weeks

Secondary ID(s)

ABR35884