Primary Ciliary Dyskinesia (PCD)MalnutritionNCT07740538
Malnutrition and Sarcopenia Among Primary Ciliary Dyskinesia
An observational study of Primary Ciliary Dyskinesia (PCD), run by Izmir Democracy University at 1 site in Turkey (Türkiye).
Trial summary
The prevalence and causes of malnutrition and sarcopenia in children and adolescents with Primary ciliary dyskinesia (PCD) have not been sufficiently elucidated. The aim of this study is to investigate the prevalence of malnutrition and sarcopenia in children and adolescents with PCD. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Primary Ciliary Dyskinesia (PCD)
- What is being tested?
- Physical assessments of individuals
- How many people
- 15 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 3 Aug 2026to 1 Jul 2028
- How long will I take part?
- 6 Months
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
- No healthy volunteers
- Child, Adult
Study population: The study will include children and adolescents aged 6-18 years with Primary Ciliary Dyskinesia (PCD).
Who may be able to join
- Unexplained neonatal respiratory distress, lateralization defect, productive cough, bronchiectasis, daily nasal congestion, and pansinusitis, as well as laboratory tests such as high-speed video microscopy, transmission electron microscopy, or genetic testing according to the European Respiratory Society diagnostic guidelines.
- Cooperative individuals,
- Individuals who have never smoked
- Willingness to participate in the study
- 1. Voluntary participation in the study
Who cannot join
- Individuals with any acute, chronic, or systemic illness other than PCD
- Individuals who smoke or are quitting smoking
- Individuals who are not willing to participate in the study
- Individuals who are uncooperative
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Turkey (Türkiye)1 site
Izmir Democracy University
Izmir, 35140
Kerim K GÖKÜSTÜN, Dr. Lecturer, 05058699032, kerimkaangokustun@hotmail.com
Gülşah BARĞI, Assoc. Dr., gulsahbargi35@gmail.com
About this study
Official title: Associations Between the Status of Malnutrition and Sarcopenia in Children and Adolescents With Primary Ciliary Dyskinesia
The prevalence and causes of malnutrition and sarcopenia in children and adolescents with Primary ciliary dyskinesia (PCD) have not been sufficiently elucidated. The aim of this study is to investigate the prevalence of malnutrition and sarcopenia in children and adolescents with PCD.
Read the detailed description
Primary ciliary dyskinesia (PCD) is an autosomal recessive disorder characterized by abnormal ciliary motility and impaired mucociliary clearance. It develops as a result of mutations in genes responsible for ciliary movement. Structural or functional abnormalities of cilia lead to chronic upper and lower respiratory tract infections, fertility problems, and organ laterality defects. The prevalence of PCD ranges from 1 in 2,000 to 1 in 40,000, varying among different ethnic groups. The disease begins to negatively affect lung function from the preschool period onward. In children with PCD, respiratory function is generally characterized by mild to moderate airway obstruction. As the disease progresses, the severity of airway obstruction increases. Therefore, regular monitoring of pulmonary function is of great importance in the management of PCD. The prevalence and causes of malnutrition and sarcopenia in children and adolescents with PCD have not been sufficiently elucidated. The aim of this study is to investigate the prevalence of malnutrition and sarcopenia in children and adolescents with PCD.
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Being tested
- Physical assessments of individuals
- Keywords
- Malnutrition, Muscle strength, Sarcopenia, Respiratory function test, Primary Ciliary Dyskinesia
- Medical subject headings
- Ciliary Motility Disorders, Malnutrition, Sarcopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Physical assessments of individualsOther
Participants will be evaluated in terms of nutritional status, anthropometric measurements (waist circumference, hip circumference, waist-to-height ratio, skinfold measurements) and body composition, pulmonary functions, anaerobic capacity, muscle strength.
| Study group | Role | What they receive |
|---|---|---|
| Patient of Primary Ciliary Dyskinesia | The study will include children and adolescents with PCD aged 6 to 18 (n=15). Receives: Other: Physical assessments of individuals |
What is being measured
The primary outcome is the main question the study is designed to answer.
Handgrip Muscle Strength in Participants
Bilateral grip strength in children and adolescents with PCD will be measured in kgF using a hand dynamometer.
Measured: From enrollment to the end of treatment at 1 year
Secondary outcomes (20)
Malnutrition risk score in Participants
The Nutritional Status and Growth Risk Screening Tool will be used to assess the risk of growth failure. The total score on this scale ranges from 0 to 5; a score of 0 indicates a low risk of malnutrition, a score of 1-3 indicates a moderate risk of malnutrition, and a score of 4-5 indicates a high risk of malnutrition.
Measured: From enrollment to the end of treatment at 1 year
Body Weight
Body weight in children and adolescents with PCD will be measured using a pediatric scale.
Measured: From enrollment to the end of treatment at 1 year
Height
In children and adolescents with PCD, height will be measured using a stadiometer.
Measured: From enrollment to the end of treatment at 1 year
Body Mass Index
For children and adolescents with PCD, the body mass index will be calculated by dividing body weight by the square of height in meters.
Measured: From enrollment to the end of treatment at 1 year
Waist circumference
Waist circumference in children and adolescents with PCD will be measured using a tape measure
Measured: From enrollment to the end of treatment at 1 year
Hip circumference
Hip circumference in children and adolescents with PCD will be measured using a tape measure
Measured: From enrollment to the end of treatment at 1 year
Waist-to-hip ratio
In children and adolescents with PCD, the waist-to-hip ratio will be calculated by dividing the waist circumference by the hip circumference.
Measured: From enrollment to the end of treatment at 1 year
Waist-to-Height ratio
In children and adolescents with PCD, the waist-to-height ratio will be calculated by dividing the waist circumference by the height.
Measured: From enrollment to the end of treatment at 1 year
Upper-middle arm circumference
In children and adolescents with PCD, upper arm circumference will be measured using a non-stretchable tape measure, without tightening it or leaving any slack.
Measured: From enrollment to the end of treatment at 1 year
Triceps skinfold thickness
In children and adolescents with PCD, triceps skinfold thickness will be measured using a skinfold caliper.
Measured: From enrollment to the end of treatment at 1 year
Body fat percentage
Body fat percentage in children and adolescents with PCD will be measured using bioelectrical impedance analysis.
Measured: From enrollment to the end of treatment at 1 year
Height by Age
The percentile values for height by age in children and adolescents with PCD will be determined using data from the CDC's website.
Measured: From enrollment to the end of treatment at 1 year
Weight by Height
The percentile values for Weight by Height in children and adolescents with PCD will be determined using data from the CDC's website.
Measured: From enrollment to the end of treatment at 1 year
Weight by Age
The percentile values for Weight by Age in children and adolescents with PCD will be determined using data from the CDC's website.
Measured: From enrollment to the end of treatment at 1 year
Body Mass Index by Age
Body Mass Index (BMI) is an anthropometric index calculated by dividing body weight in kilograms by the square of height in meters. Body Mass Index by Age (BMI-for-Age) is the body mass index (BMI) adjusted for age and sex and interpreted using age- and sex-specific growth reference charts.The percentile values for Body Mass Index by Age in children and adolescents with PCD will be determined using data from the CDC's website.
Measured: From enrollment to the end of treatment at 1 year
Forced Expiratory Volume at One Second
Forced Expiratory Volume at One Second is the volume of air that an individual can forcibly exhale during the first second of a maximal forced expiration following a full inspiration. It is typically expressed in liters or as a percentage of the predicted value (% predicted).
Measured: From enrollment to the end of treatment at 1 year
Forced Vital Capacity
Forced Vital Capacity is the total volume of air that an individual can forcibly exhale after taking a maximal inspiration. It is typically expressed in liters (L) or as a percentage of the predicted value (% predicted).
Measured: From enrollment to the end of treatment at 1 year
Forced Expiratory Volume at One Secondto Forced Vital Capacity Ratio
Forced Expiratory Volume at One Second to Forced Vital Capacity Ratio is the proportion of the forced vital capacity that is exhaled during the first second of a forced expiration following a maximal inspiration. It is expressed as a percentage.
Measured: From enrollment to the end of treatment at 1 year
Peak Expiratory Flow
Peak Expiratory Flow is the maximum expiratory flow rate achieved during a forced expiration following a maximal inspiration. It is typically expressed in liters per minute.
Measured: From enrollment to the end of treatment at 1 year
Forced Expiratory Flow at 25-75% of Forced Vital Capacity
Forced Expiratory Flow at 25-75% of Forced Vital Capacity is the average forced expiratory flow measured during the middle 50% of the forced vital capacity maneuver, specifically between 25% and 75% of the exhaled volume. It is typically expressed in liters per second.
Measured: From enrollment to the end of treatment at 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2026
- Study start3 Aug 2026 (estimated)
- Primary completion1 Jul 2027 (estimated)
- Study completion1 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Primary Ciliary Dyskinesia (PCD)Studies of the same condition, open and under way.
- 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 only
- Time perspective
- Cross-sectional
- Enrolment
- 15 participants (estimated)
- Target duration
- 6 Months
Sponsor and investigators
- Lead sponsor
- Izmir Democracy University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Kerim Kaan Göküstün, Izmir Democracy University
- Investigators
- Kerim K Göküstün, Dr. Lecturer, Study director, Izmir Democracy UniversityGülşah BARĞI, Assoc. Dr., Principal investigator, Izmir Democracy UniversityAybüke Sena DEMİR, MSc., Principal investigator, Izmir Democracy UniversityEce OCAK, Dr., Principal investigator, University of Health science
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07740538
- Sponsor's protocol ID
- Primary Ciliary Dyskinesia
- Acronym
- Malnutrition
- Status last verified
- Jul 2026
- First submitted
- 23 Jul 2026
- Last updated
- 31 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Despotes KA, Zariwala MA, Davis SD, Ferkol TW. Primary Ciliary Dyskinesia: A Clinical Review. Cells. 2024 Jun 4;13(11):974. doi: 10.3390/cells13110974.
PubMed 38891105McCoy J, Gatt D, Shaw M, Solomon M, Kritzinger F, Ratjen F. The Impact of Nutritional Status on Lung Function Trajectories in Pediatric Patients With Primary Ciliary Dyskinesia. Pediatr Pulmonol. 2026 Apr;61(4):e71634. doi: 10.1002/ppul.71634.
PubMed 42001405Karavasiloglou N, Gkatzou V, Fernandez-Rodriguez A, Schwartz V, Goutaki M. Nutrition and growth of primary ciliary dyskinesia patients: a systematic review. Eur Respir Rev. 2026 May 27;35(180):260024. doi: 10.1183/16000617.0024-2026. Print 2026 Apr.
PubMed 42203237
Study at a glance
From the registry record, last updated by the sponsor on 31 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Associations Between the Status of Malnutrition and Sarcopenia in Children and Adolescents With Primary Ciliary Dyskinesia
- ClinicalTrials.gov ID
- NCT07740538
- Acronym / sponsor ID
- Malnutrition / Primary Ciliary Dyskinesia
- Conditions
- Primary Ciliary Dyskinesia (PCD)
- Keywords
- Malnutrition, Muscle strength, Sarcopenia, Respiratory function test, Primary Ciliary Dyskinesia
- Study type
- Observational
- Recruitment status
- Not yet recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Izmir Democracy UniversityUniversity, hospital or other
- Study start
- 3 Aug 2026 (estimated)
- Primary completion
- 1 Jul 2027 (estimated)
- Study completion
- 1 Jul 2028 (estimated)
- First posted
- 31 Jul 2026 (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.
The prevalence and causes of malnutrition and sarcopenia in children and adolescents with Primary ciliary dyskinesia (PCD) have not been sufficiently elucidated. The aim of this study is to investigate the prevalence of malnutrition and sarcopenia in children and adolescents with PCD.
Detailed description
Primary ciliary dyskinesia (PCD) is an autosomal recessive disorder characterized by abnormal ciliary motility and impaired mucociliary clearance. It develops as a result of mutations in genes responsible for ciliary movement. Structural or functional abnormalities of cilia lead to chronic upper and lower respiratory tract infections, fertility problems, and organ laterality defects. The prevalence of PCD ranges from 1 in 2,000 to 1 in 40,000, varying among different ethnic groups. The disease begins to negatively affect lung function from the preschool period onward. In children with PCD, respiratory function is generally characterized by mild to moderate airway obstruction. As the disease progresses, the severity of airway obstruction increases. Therefore, regular monitoring of pulmonary function is of great importance in the management of PCD. The prevalence and causes of malnutrition and sarcopenia in children and adolescents with PCD have not been sufficiently elucidated. The aim of this study is to investigate the prevalence of malnutrition and sarcopenia in children and adolescents with PCD.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Target follow-up
- 6 Months
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient of Primary Ciliary Dyskinesia | – | Other: Physical assessments of individualsThe study will include children and adolescents with PCD aged 6 to 18 (n=15). |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- The study will include children and adolescents aged 6-18 years with Primary Ciliary Dyskinesia (PCD).
Key inclusion criteria (5 listed)
- Unexplained neonatal respiratory distress, lateralization defect, productive cough, bronchiectasis, daily nasal congestion, and pansinusitis, as well as laboratory tests such as high-speed video microscopy, transmission electron microscopy, or genetic testing according to the European Respiratory Society diagnostic guidelines.
- Cooperative individuals,
- Individuals who have never smoked
- Willingness to participate in the study
- 1. Voluntary participation in the study
Key exclusion criteria (4 listed)
- Individuals with any acute, chronic, or systemic illness other than PCD
- Individuals who smoke or are quitting smoking
- Individuals who are not willing to participate in the study
- Individuals who are uncooperative
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Physical assessments of individualsParticipants will be evaluated in terms of nutritional status, anthropometric measurements (waist circumference, hip circumference, waist-to-height ratio, skinfold measurements) and body composition, pulmonary functions, anaerobic capacity, muscle strength. | Other | Patient of Primary Ciliary Dyskinesia |
Primary outcome
Handgrip Muscle Strength in Participants
Time frame: From enrollment to the end of treatment at 1 year
Bilateral grip strength in children and adolescents with PCD will be measured in kgF using a hand dynamometer.
20 secondary outcomes
Malnutrition risk score in Participants
Time frame: From enrollment to the end of treatment at 1 year
Body Weight
Time frame: From enrollment to the end of treatment at 1 year
Height
Time frame: From enrollment to the end of treatment at 1 year
Body Mass Index
Time frame: From enrollment to the end of treatment at 1 year
Waist circumference
Time frame: From enrollment to the end of treatment at 1 year
Hip circumference
Time frame: From enrollment to the end of treatment at 1 year
Waist-to-hip ratio
Time frame: From enrollment to the end of treatment at 1 year
Waist-to-Height ratio
Time frame: From enrollment to the end of treatment at 1 year
Upper-middle arm circumference
Time frame: From enrollment to the end of treatment at 1 year
Triceps skinfold thickness
Time frame: From enrollment to the end of treatment at 1 year
Body fat percentage
Time frame: From enrollment to the end of treatment at 1 year
Height by Age
Time frame: From enrollment to the end of treatment at 1 year
Weight by Height
Time frame: From enrollment to the end of treatment at 1 year
Weight by Age
Time frame: From enrollment to the end of treatment at 1 year
Body Mass Index by Age
Time frame: From enrollment to the end of treatment at 1 year
Forced Expiratory Volume at One Second
Time frame: From enrollment to the end of treatment at 1 year
Forced Vital Capacity
Time frame: From enrollment to the end of treatment at 1 year
Forced Expiratory Volume at One Secondto Forced Vital Capacity Ratio
Time frame: From enrollment to the end of treatment at 1 year
Peak Expiratory Flow
Time frame: From enrollment to the end of treatment at 1 year
Forced Expiratory Flow at 25-75% of Forced Vital Capacity
Time frame: From enrollment to the end of treatment at 1 year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jul 2026.
- Background: PubMed 38891105Despotes KA, Zariwala MA, Davis SD, Ferkol TW. Primary Ciliary Dyskinesia: A Clinical Review. Cells. 2024 Jun 4;13(11):974. doi: 10.3390/cells13110974.
- Background: PubMed 42001405McCoy J, Gatt D, Shaw M, Solomon M, Kritzinger F, Ratjen F. The Impact of Nutritional Status on Lung Function Trajectories in Pediatric Patients With Primary Ciliary Dyskinesia. Pediatr Pulmonol. 2026 Apr;61(4):e71634. doi: 10.1002/ppul.71634.
- Background: PubMed 42203237Karavasiloglou N, Gkatzou V, Fernandez-Rodriguez A, Schwartz V, Goutaki M. Nutrition and growth of primary ciliary dyskinesia patients: a systematic review. Eur Respir Rev. 2026 May 27;35(180):260024. doi: 10.1183/16000617.0024-2026. Print 2026 Apr.
- Individual participant dataSharing planned: No
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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.
Kerim K Göküstün, Dr. Lecturer study director
Izmir Democracy University
Gülşah BARĞI, Assoc. Dr. principal investigator
Izmir Democracy University
Aybüke Sena DEMİR, MSc. principal investigator
Izmir Democracy University
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 (PCD), run by Izmir Democracy 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 5 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: opening soon, as of the sponsor's last update on 31 Jul 2026. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Kerim Kaan K GÖKÜSTÜN, Dr. Lecturer (+90 232 299 07 39, kerimkaangokustun@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Handgrip Muscle Strength in Participants (time frame: From enrollment to the end of treatment at 1 year). 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 NCT07740538, which also lists 3 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 31 Jul 2026.