Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Daily Living Activities and Exercise Capacity in Lipid Storage Diseases

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03875105
Recruitment Status : Not yet recruiting
First Posted : March 14, 2019
Last Update Posted : August 7, 2019
Sponsor:
Collaborator:
Biruni University
Information provided by (Responsible Party):
Goksen Kuran Aslan, Istanbul University

Brief 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.

Condition or disease
Lipid Storage Disease

Detailed 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.

Layout table for study information
Study Type : Observational [Patient Registry]
Estimated Enrollment : 25 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 1 Day
Official Title: The Investigation of Daily Living Activities and Exercise Capacity in Lipid Storage Diseases
Estimated Study Start Date : September 1, 2019
Estimated Primary Completion Date : January 1, 2020
Estimated Study Completion Date : April 1, 2020

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. 6 minutes walking test [ Time Frame: 1 day ]
    Exercise capacity will be evaluated with 6 minutes walking test


Secondary Outcome Measures :
  1. Activity limitation (ACTIVLIM) questionnaire [ Time Frame: 1 day ]
    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.

  2. Rivermead mobility index [ Time Frame: 1 day ]
    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.

  3. Hand held dinamometer [ Time Frame: 1 day ]
    Hand held dinamometer will be used for the measurement of upper and lower muscle strength.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
25 patients who were diagnosed with Lipid Storage Disease at Istanbul University Istanbul Medical Faculty Neurology and Chest Diseases Departments will be invited to the study. The study will be continued in Istanbul University, Istanbul Faculty of Medicine, Department of Chest Diseases.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03875105


Contacts
Layout table for location contacts
Contact: Goksen Kuran Aslan, Assoc.Prof +90 (212) 414 15 28 ext 40167 goksenkuran@yahoo.com

Locations
Layout table for location information
Turkey
Istanbul University Not yet recruiting
Istanbul, Turkey
Contact: Goksen Kuran Aslan, Assoc.Prof.    +90 (212) 414 15 28 ext 40167    goksenkuran@yahoo.com   
Sub-Investigator: Buket Akinci, Assist.Prof.         
Sub-Investigator: Esen Kiyan, Prof.Dr.         
Sub-Investigator: Hacer Durmus Tekce, Assoc.Prof.         
Sponsors and Collaborators
Istanbul University
Biruni University
Investigators
Layout table for investigator information
Principal Investigator: Goksen Kuran Aslan, Assoc.Prof. Istanbul University-Cerrahpasa

Additional Information:

Publications of Results:
Layout table for additonal information
Responsible Party: Goksen Kuran Aslan, Associated Proffesor, Istanbul University
ClinicalTrials.gov Identifier: NCT03875105     History of Changes
Other Study ID Numbers: 2018/24-13
First Posted: March 14, 2019    Key Record Dates
Last Update Posted: August 7, 2019
Last Verified: August 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Goksen Kuran Aslan, Istanbul University:
exercise capacity
activities of daily living
Additional relevant MeSH terms:
Layout table for MeSH terms
Lipidoses
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipid Metabolism Disorders