Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

Lipid Storage Disease Clinical Trial

Official title:

The Investigation of Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT03875105
• Other study ID #: 2018/24-13
• Status: Not yet recruiting
• Start date: September 2, 2023
• Est. completion date: November 5, 2024

Study information

• Verified date: August 2023
• Source: Istanbul University
• Contact: Goksen Kuran Aslan, Assoc.Prof
• Phone: +90 (212) 414 15 28
• Email: goksenkuran[@]yahoo.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Lipid Storage Diseases (LSD) is a group of metabolic diseases that are manifested by exercise intolerance or muscle weakness, sometimes accompanied by systemic findings, which develop in some stages of muscle contraction due to necessary lipid transfer or destruction pathways. Exercise intolerance in LSD can limit not only physical activity but also daily activities. In the literature, no study evaluated the activities of daily living in patients with LSD. The aim of this study was to investigate the effect of exercise capacity and daily living activities on the clinical features of LSD patients.

Description:

Pulmonary functions are affected negatively in Lipid Storage Diseases (LSD). It was shown that respiratory muscle strength and peak cough flow decreased in these cases. Other respiratory system problems can be listed as; diaphragmatic dysfunction, hypoxemia, and hypercapnia. Routine evaluation of respiratory function in LSD is recommended. A limited number of studies have shown that exercise tolerance is limited by the blockage of fat oxidation on the basis of muscle weakness and fatigue in LSD. Although the assessment of exercise capacity is also an important indicator of heart-lung capacity monitoring, exercise capacity is not routinely measured in patients with LSD. Exercise intolerance can limit not only physical activity but also daily activities. In the literature, the investigator's did not find any study evaluating the activities of daily living in patients with LSD. The aim of this study was to investigate the effect of exercise capacity and daily living activities on the clinical features of LSD patients.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 25
• Est. completion date: November 5, 2024
• Est. primary completion date: September 4, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

Inclusion Criteria:

• Being diagnosed with Lipid storage disease clinically and histopathologically.

Exclusion Criteria:

• Severe pulmonary parenchymal disease,

• Cardiac problems

• Renal or hepatic dysfunction

• Uncontrolled hypothyroidism

• Acute infection

• Malignancy

Related Conditions & MeSH terms

• Lipid Storage Disease
• Metabolic Diseases

Locations

Country	Name	City	State
Turkey	Istanbul University	Istanbul

Sponsors (2)

Lead Sponsor	Collaborator
Istanbul University	Biruni University

Country where clinical trial is conducted

Turkey, 

References & Publications (1)

Bingol Z, Tekce HD, Sagcan G, Serdaroglu P, Kiyan E. Pulmonary functions and sleep-related breathing disorders in lipid storage disease. Sleep Breath. 2018 Dec;22(4):1137-1142. doi: 10.1007/s11325-018-1647-1. Epub 2018 Mar 1. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	6 minutes walking test	Exercise capacity will be evaluated with 6 minutes walking test	1 day
Secondary	Activity limitation (ACTIVLIM) questionnaire	Activities of daily living will be evaluated Activlim questionnaire. The questionnaire is consists of 22 questions. Minimum score is 0 and total score is 88. Higher scores indicate worse conditions.	1 day
Secondary	Rivermead mobility index	Mobility will be evaluated with Rivermead mobility index. The questionnaire consists of 14 questions. Minimum score is 0, maximum score is 15. Higher scores indicate better conditions.	1 day
Secondary	Hand held dinamometer	Hand held dinamometer will be used for the measurement of upper and lower muscle strength.	1 day