Primary Ciliary DyskinesiaNCT03370029
Respiratory Muscle Strength, Exercise Capacity and Physical Activity Levels in Children Primary Ciliary Dyskinesia
An observational study of Primary Ciliary Dyskinesia, run by Gazi University at 1 site in Turkey (Türkiye).
Trial summary
Primary ciliary dyskinesia is an autosomal recessive disorder characterized by abnormal ciliary movement and disrupted mucociliary clearance. In uncleaned airways, microorganisms and respiratory irritants cause inflammation and infection. Read more about this study
- Who is it for?
- Ages 6 to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Primary Ciliary Dyskinesia
- How many people
- 26 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- Oct 2016to 30 Jun 2018
- 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 6 to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Study population: Out- patients and healthy volunteers
Who may be able to join
- Diagnosed primary ciliary dyskinesia patients,
- 6-18 years of age,
- under standard medications,
- stable patients without exacerbation or infection
Who cannot join
- having cognitive disorder,
- orthopedic or neurological disease with a potential to affect functional capacity,
- acute infections or pneumonia,
- problems which may prevent evaluating such as visual problems
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
Gazi University Faculty of Health Sciences Department of Physical Therapy and Rehabilitation
Yenimahalle
About this study
Official title: Comparison of Respiratory Muscle Strength, Exercise Capacity and Physical Activity Levels in Children With Primary Ciliary Dyskinesia and Healthy Controls
Primary ciliary dyskinesia is an autosomal recessive disorder characterized by abnormal ciliary movement and disrupted mucociliary clearance. In uncleaned airways, microorganisms and respiratory irritants cause inflammation and infection. Permanent rhinitis and chronic sputum cough are typical features in primary ciliary dyskinesia patients. Primary ciliary dyskinesia is a disease that threatens lung function from pre-school age. One of the main causes of respiratory muscle weakness in chronic lung diseases diseases is worsening of lung function. Such a weakness causes alveolar hypoventilation, microatelectasis, reduction of the cough strength .The cough strength is important for airway cleaning.
Exercise capacity is affected in chronic lung diseases. Assessment of exercise capacity in chronic lung diseases is prognostically important. Reduced exercise capacity and pulmonary function in PCD cause decrease in physical activity level. PCD patients have low quality of life and early recognition has been found to affect the quality of life positively. Children with chronic illness have higher level of depression than healthy children.
In literature, no study investigated respiratory muscle strength, exercise capacity and physical activity PCD patients. Therefore, the investigators aimed to compare aforementioned outcomes in PCD patients and healthy controls.
Read the detailed description
According to sample size calculation 26 diagnosed primary ciliary dyskinesia patients and 26 healthy individuals will be included. Respiratory muscle strength, anaerobic and aerobic exercise capacity, physical activity, pulmonary functions, peripheral muscle strength, cough strength, respiratory muscle endurance, activity dyspnea perception, quality of life and depression will be evaluated. Primary outcome measurements are respiratory muscle strength, exercise capacity, physical activity; secondary outcomes are pulmonary functions, peripheral muscle strength, cough strength, respiratory muscle endurance, activity dyspnea perception, quality of life and depression.
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- Exercise capacity, Respiratory muscle strength, Physical inactivity, Quality of life
- Medical subject headings
- Ciliary Motility Disorders, Sedentary Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Primary ciliary dyskinesia patients | Primary ciliary dyskinesia patients will be included in study. Inclusion and exclusion criteria were considered. | |
| Healthy individuals | Those without diagnosed chronic disease will be included in study. Inclusion and exclusion criteria were considered. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Respiratory muscle strength
Evaluated using mouth pressure device
Measured: first day
Functional exercise capacity
Evaluated using 6-minute walking test
Measured: first day
Anaerobic exercise capacity
Evaluated using 3-minute step test
Measured: first day
Physical activity
Evaluated using a metabolic holter
Measured: first day
Secondary outcomes (8)
Pulmonary functions
Evaluated using spirometer
Measured: first day
Muscle strength
Evaluated using hand-held dynamometer for peripheral muscles (shoulder abduction, elbow flexion, knee extension, hand grip strength)
Measured: second day
Cough strength
Evaluated using PEFmeter
Measured: second day
Respiratory muscle endurance
Evaluated using respiratory muscle trainer (POWERbreathe)
Measured: second day]
Activity dyspnea
Evaluated using Modified Borg Scale (0-10 scores, higher values represent a worse outcome)
Measured: first day
Generic Quality of life
Cystic Fibrosis Questionnaire Revised (CFQ-R) Turkish version (Scores range from 0 to 100, with higher scores indicating better health. 9 Quality of life domains:
Physical, role/school, vitality, emotion, social, body image, eating, treatment burden, health perceptions.3 symptom scales:
Weight, respiratory, and digestion.Number of items in CFQ-R CFQ-R Teen/Adult: 50. CFQ-R Parent: 44. CFQ-R Child: 35.)
Measured: second day
Disease- specific quality of life
Health-related quality-of-life questionnaires-Primary Ciliary Dyskinesia ( PCD-QOL) Turkish version ( PCD-QOL questionnaire developed different domains for different age groups: pediatric, adolescent, and adult patients as well as parents. These domains mainly evaluate physical, emotional, and social aspects of PCD related to QOL. Moreover, there are different domains for various symptoms at different age groups. The total numbers of items in the questionnaires are 37 in the questionnaire for children, 43 in the one for adolescents, 49 in the one for adults, 41 in the parents' questionnaire.
Measured: second day
Depression
Children's Depression Inventory Turkish Version (Children's Depression Inventory is a 27-item, self-rated, symptom-oriented scale.
Measured: second day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Dec 2017
- Study startOct 2016
- Primary completion25 Feb 2018
- Study completion30 Jun 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Primary Ciliary DyskinesiaStudies of the same condition, open and under way.
- More trials run by Gazi 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
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gazi University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Meral Boşnak Güçlü, Gazi University
- Investigators
- Merve Fırat, Pt, Study chair, Research assistantMeral Boşnak Güçlü, Pt,Phd, Study director, Associate professorTuğba Şişmanlar Eyüboğlu, MD, Principal investigator, Medical doctorAyşe Tana Aslan, MD,Phd, Principal investigator, Professor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03370029
- Sponsor's protocol ID
- Gazi University5
- Status last verified
- Feb 2019
- First submitted
- 10 Nov 2017
- Last updated
- 15 Feb 2019
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No: There is not a plan to make individual participant data but when the statistical analyses of all data are made, all results will be shared.
Publications
Firat M, Bosnak-Guclu M, Sismanlar-Eyuboglu T, Aslan AT. Aerobic exercise capacity, cough strength, posture, and depression in primary ciliary dyskinesia. Pediatr Pulmonol. 2023 Jan;58(1):73-80. doi: 10.1002/ppul.26158. Epub 2022 Sep 28.
PubMed 36114723Firat M, Bosnak-Guclu M, Sismanlar-Eyuboglu T, Tana-Aslan A. Respiratory muscle strength, exercise capacity and physical activity in patients with primary ciliary dyskinesia: A cross-sectional study. Respir Med. 2022 Jan;191:106719. doi: 10.1016/j.rmed.2021.106719. Epub 2021 Dec 17.
PubMed 34952415
Study at a glance
From the registry record, last updated by the sponsor on 15 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Respiratory Muscle Strength, Exercise Capacity and Physical Activity Levels in Children With Primary Ciliary Dyskinesia and Healthy Controls
- ClinicalTrials.gov ID
- NCT03370029
- Acronym / sponsor ID
- Gazi University5
- Conditions
- Primary Ciliary Dyskinesia
- Keywords
- Exercise capacity, Respiratory muscle strength, Physical inactivity, Quality of life
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Gazi UniversityUniversity, hospital or other
- Study start
- Oct 2016 (actual)
- Primary completion
- 25 Feb 2018 (actual)
- Study completion
- 30 Jun 2018 (actual)
- First posted
- 12 Dec 2017 (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.
Primary ciliary dyskinesia is an autosomal recessive disorder characterized by abnormal ciliary movement and disrupted mucociliary clearance. In uncleaned airways, microorganisms and respiratory irritants cause inflammation and infection. Permanent rhinitis and chronic sputum cough are typical features in primary ciliary dyskinesia patients. Primary ciliary dyskinesia is a disease that threatens lung function from pre-school age. One of the main causes of respiratory muscle weakness in chronic lung diseases diseases is worsening of lung function. Such a weakness causes alveolar hypoventilation, microatelectasis, reduction of the cough strength .The cough strength is important for airway cleaning.
Exercise capacity is affected in chronic lung diseases. Assessment of exercise capacity in chronic lung diseases is prognostically important. Reduced exercise capacity and pulmonary function in PCD cause decrease in physical activity level. PCD patients have low quality of life and early recognition has been found to affect the quality of life positively. Children with chronic illness have higher level of depression than healthy children.
In literature, no study investigated respiratory muscle strength, exercise capacity and physical activity PCD patients. Therefore, the investigators aimed to compare aforementioned outcomes in PCD patients and healthy controls.
Detailed description
According to sample size calculation 26 diagnosed primary ciliary dyskinesia patients and 26 healthy individuals will be included. Respiratory muscle strength, anaerobic and aerobic exercise capacity, physical activity, pulmonary functions, peripheral muscle strength, cough strength, respiratory muscle endurance, activity dyspnea perception, quality of life and depression will be evaluated. Primary outcome measurements are respiratory muscle strength, exercise capacity, physical activity; secondary outcomes are pulmonary functions, peripheral muscle strength, cough strength, respiratory muscle endurance, activity dyspnea perception, quality of life and depression.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Primary ciliary dyskinesia patients | – | –Primary ciliary dyskinesia patients will be included in study. Inclusion and exclusion criteria were considered. |
| Healthy individuals | – | –Those without diagnosed chronic disease will be included in study. Inclusion and exclusion criteria were considered. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Out- patients and healthy volunteers
Key inclusion criteria (4 listed)
- Diagnosed primary ciliary dyskinesia patients,
- 6-18 years of age,
- under standard medications,
- stable patients without exacerbation or infection
Key exclusion criteria (4 listed)
- having cognitive disorder,
- orthopedic or neurological disease with a potential to affect functional capacity,
- acute infections or pneumonia,
- problems which may prevent evaluating such as visual problems
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
Respiratory muscle strength
Time frame: first day
Evaluated using mouth pressure device
Functional exercise capacity
Time frame: first day
Evaluated using 6-minute walking test
Anaerobic exercise capacity
Time frame: first day
Evaluated using 3-minute step test
Physical activity
Time frame: first day
Evaluated using a metabolic holter
8 secondary outcomes
Pulmonary functions
Time frame: first day
Muscle strength
Time frame: second day
Cough strength
Time frame: second day
Respiratory muscle endurance
Time frame: second day]
Activity dyspnea
Time frame: first day
Generic Quality of life
Time frame: second day
Disease- specific quality of life
Time frame: second day
Depression
Time frame: second day
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Feb 2019.
- Derived from this study: PubMed 36114723Firat M, Bosnak-Guclu M, Sismanlar-Eyuboglu T, Aslan AT. Aerobic exercise capacity, cough strength, posture, and depression in primary ciliary dyskinesia. Pediatr Pulmonol. 2023 Jan;58(1):73-80. doi: 10.1002/ppul.26158. Epub 2022 Sep 28.
- Derived from this study: PubMed 34952415Firat M, Bosnak-Guclu M, Sismanlar-Eyuboglu T, Tana-Aslan A. Respiratory muscle strength, exercise capacity and physical activity in patients with primary ciliary dyskinesia: A cross-sectional study. Respir Med. 2022 Jan;191:106719. doi: 10.1016/j.rmed.2021.106719. Epub 2021 Dec 17.
- Related InfoLink provided by the sponsor.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No. There is not a plan to make individual participant data but when the statistical analyses of all data are made, all results will be shared.
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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.
Merve Fırat, Pt study chair
Research assistant
Meral Boşnak Güçlü, Pt,Phd study director
Associate professor
Tuğba Şişmanlar Eyüboğlu, MD principal investigator
Medical doctor
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 Primary Ciliary Dyskinesia, run by Gazi 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 4 inclusion and 4 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: finished, as of the sponsor's last update on 15 Feb 2019. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Respiratory muscle strength (time frame: first day). The study lists 4 primary outcomes in all. 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 NCT03370029, which also lists 2 linked publications.
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 15 Feb 2019.