Lipid Storage DiseaseNCT03875105
Daily Living Activities and Exercise Capacity in Lipid Storage Diseases
An observational study of Lipid Storage Disease, run by Istanbul University at 1 site in Turkey (Türkiye).
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Lipid Storage Disease
- How many people
- 25 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 2 Sep 2023to 5 Nov 2024
- How long will I take part?
- 1 Day
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
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.
Who may be able to join
- Being diagnosed with Lipid storage disease clinically and histopathologically.
Who cannot join
- Severe pulmonary parenchymal disease,
- Cardiac problems
- Renal or hepatic dysfunction
- Uncontrolled hypothyroidism
- Acute infection
- Malignancy
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Istanbul University
Istanbul
Goksen Kuran Aslan, Assoc.Prof., +90 (212) 414 15 28 ext. 40167, goksenkuran@yahoo.com
Buket Akinci, Assist.Prof.
About this study
Official title: The Investigation of Daily Living Activities and Exercise Capacity in Lipid Storage Diseases
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.
Read the 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.
- Conditions
- Lipid Storage Disease
- Keywords
- exercise capacity, activities of daily living
- Medical subject headings
- Lipidoses
What is being measured
The primary outcome is the main question the study is designed to answer.
6 minutes walking test
Exercise capacity will be evaluated with 6 minutes walking test
Measured: 1 day
Secondary outcomes (3)
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.
Measured: 1 day
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.
Measured: 1 day
Hand held dinamometer
Hand held dinamometer will be used for the measurement of upper and lower muscle strength.
Measured: 1 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2019
- Study start2 Sep 2023 (estimated)
- Primary completion4 Sep 2024 (estimated)
- Study completion5 Nov 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Lipid Storage DiseaseStudies of the same condition, open and under way.
- More trials run by Istanbul UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (estimated)
- Target duration
- 1 Day
Sponsor and investigators
- Lead sponsor
- Istanbul University
- Sponsor type
- University, hospital or other
- Collaborators
- Biruni University
- Responsible party
- Principal investigator, Goksen Kuran Aslan, Istanbul University
- Investigators
- Goksen Kuran Aslan, Assoc.Prof., Principal investigator, Istanbul University - Cerrahpasa
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03875105
- Sponsor's protocol ID
- 2018/24-13
- Status last verified
- Aug 2023
- First submitted
- 11 Mar 2019
- Last updated
- 16 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
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.
PubMed 29497949
Study at a glance
From the registry record, last updated by the sponsor on 16 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Investigation of Daily Living Activities and Exercise Capacity in Lipid Storage Diseases
- ClinicalTrials.gov ID
- NCT03875105
- Acronym / sponsor ID
- 2018/24-13
- Conditions
- Lipid Storage Disease
- Keywords
- exercise capacity, activities of daily living
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Istanbul UniversityUniversity, hospital or other
- Collaborators
- Biruni University
- Study start
- 2 Sep 2023 (estimated)
- Primary completion
- 4 Sep 2024 (estimated)
- Study completion
- 5 Nov 2024 (estimated)
- First posted
- 14 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 1 Day
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- 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.
Key inclusion criteria (1 listed)
- Being diagnosed with Lipid storage disease clinically and histopathologically.
Key exclusion criteria (6 listed)
- Severe pulmonary parenchymal disease,
- Cardiac problems
- Renal or hepatic dysfunction
- Uncontrolled hypothyroidism
- Acute infection
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
6 minutes walking test
Time frame: 1 day
Exercise capacity will be evaluated with 6 minutes walking test
3 secondary outcomes
Activity limitation (ACTIVLIM) questionnaire
Time frame: 1 day
Rivermead mobility index
Time frame: 1 day
Hand held dinamometer
Time frame: 1 day
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Aug 2023.
- Results publication: PubMed 29497949Bingol 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Goksen Kuran Aslan, Assoc.Prof. principal investigator
Istanbul University - Cerrahpasa
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lipid Storage Disease, run by Istanbul University at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Goksen Kuran Aslan, Assoc.Prof (+90 (212) 414 15 28 ext. 40167, goksenkuran@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
6 minutes walking test (time frame: 1 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03875105, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Aug 2023.