Primary Ciliary DyskinesiaBHRNCT07288827
Examining Bronchial Hyperresponsiveness in Primary Ciliary Dyskinesia
A study testing Lung Function Testing, Phlebotomy and Allergy skin prick testing in people with Primary Ciliary Dyskinesia and Healthy, run by Indiana University at 1 site in United States.
Trial summary
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness". Read more about this study
- Who is it for?
- Ages 6 years and olderHealthy volunteers can take part
- Conditions the study is for
- Primary Ciliary Dyskinesia, Healthy
- What is being tested?
- Lung Function Testing, Phlebotomy and Allergy skin prick testing
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 4 May 2023to 30 Apr 2028
- Phase
- Interventional
- 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 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- Confirmed diagnosis of PCD per standard diagnostic criteria4 and positive genetics
- Age greater than or equal to 6 years (no upper age limit)
- Any gender or race
- Able to perform pulmonary function testing (historical documentation of reversibility will be accepted)
Who cannot join
- history of current pneumothorax
- inability to perform pulmonary function testing
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Riley Hospital for Children
Indianapolis, Indiana, 46202
Fanmuyi Yang, PhD
About this study
Official title: A Multi-center Study Examining Bronchial Hyperresponsiveness in Primary Ciliary Dyskinesia
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness".
Read the detailed description
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness". This is when some of the air tubes ("bronchi") are very sensitive ("hyperresponsive") to certain things in the environment such as pollen, mold, pet dander, changes in temperature, and viruses. Children with PCD are often prescribed daily inhaled steroids to help make their air tubes less sensitive, but there are few research studies showing that children with PCD really need this medication.
In this study, the investigators will simulate the above triggers for sensitive air tubes with a medication called methacholine. Checking how fast children can breathe out after giving methacholine is sometimes done when doctors think a child may have asthma, a condition where the breathing tubes are very sensitive. By measuring the participant's breathing before and after giving this medication, the investigators will see whether their air tubes are sensitive, or "hyperresponsive". It is possible that methacholine could make the participants temporarily feel short of breath or start wheezing. To avoid this, the investigators will start by giving a small dose of methacholine and measuring the participants breathing, then give larger amounts of methacholine until the investigators see certain changes in the participants breathing or until the largest dose allowed is reached. If the participant starts to have symptoms such as shortness of breath, chest tightness, or wheezing, the investigators will give them a medicine called albuterol which immediately opens the air tubes and makes the symptoms go away. Any discomfort caused by the methacholine is temporary and there are no long-term effects associated with using it.
If the participants agree to participate in the study, they will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, they will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw.
The investigators will also collect information about the participants from your medical record, including demographic information, medical history, and disease history. If they have completed pulmonary function testing (testing that shows how well lungs are working) in the past, they may only be eligible for Visit 1.
For scheduling convenience, visits 1 and 2 may occur on the same day. The participants will be in this study up to one month if they participate in visits 1 and 2, and up to five months if they are eligible for visit 3.
- Conditions
- Primary Ciliary Dyskinesia, Healthy
- Being tested
- Lung Function Testing, Phlebotomy, Allergy skin prick testing, Methacholine Challenge
- Keywords
- Primary Ciliary Dyskinesia, bronchial hyperresponsiveness
- Medical subject headings
- Ciliary Motility Disorders, Bronchial Hyperreactivity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Lung Function TestingProcedure or surgery
Also called spirometry
will include baseline spirometry (pre- and post- max bronchodilator). All testing will be done according to American Thoracic Society/European Respiratory Society guidelines. Participants will be asked to refrain from taking any asthma medications, including inhaled corticosteroids, short- and long-acting bronchodilators, leukotriene receptor antagonists, and long-acting muscarinic antagonists, for 24 hours prior to any spirometry. This activity will take place at a clinical research center at the respective participating institution.
PhlebotomyProcedure or surgery
Also called blood draw
will be obtained at visit 2. Up to ten (10) ml of blood will be collected for measurement of serum biomarkers of atopy, based on whether participants prefer to receive an allergy skin prick test or have antigen-specific IgE levels tested. In the event a subject refuses phlebotomy, historical results up to one year old may be used in lieu of prospective results. Any remaining blood samples will be banked either for use in future studies or in the event that additional serum biomarkers are added to this study.
Allergy skin prick testingProcedure or surgery
may be completed at visit 2. Subjects will be instructed to withhold first-generation antihistamines for 3 days and second-generation antihistamines for 7 days prior to the test. If patients prefer to have serum antigen-specific IgE levels run with the required serum biomarkers of atopy, then skin prick testing will be omitted.
Methacholine ChallengeProcedure or surgery
Also called MCT
If the subject does not demonstrate a bronchodilator response in FEV1 of 10% or greater, and does not have a historical MCT on file, MCT will be performed. Following inhalation of saline, methacholine (MCh) will be inhaled in quadrupling concentrations starting with 0.0625 mg/ml and continuing until the MCh concentration required for FEV1 to decrease by 20% from baseline (PD20) is achieved or a maximum MCh concentration of 16 mg/ml is inhaled.
| Study group | Role | What they receive |
|---|---|---|
| Subjects with Primary Ciliary Dyskinesia | Experimental | Subjects will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, subjects will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw. Receives: Procedure: Lung Function Testing; Procedure: Phlebotomy; Procedure: Allergy skin prick testing; Procedure: Methacholine Challenge |
| Healthy Subjects | Experimental | Subjects will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, subjects will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw. Receives: Procedure: Lung Function Testing; Procedure: Phlebotomy; Procedure: Allergy skin prick testing; Procedure: Methacholine Challenge |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bronchial Hyperresponsiveness prevalence
Examine how many subjects with PCD have bronchial hyperresponsiveness as evidenced by an increase in FEV1 of 10% or greater during pre- and post- max bronchodilator test.
Measured: From spirometry baseline measurement (Visit 2) to completion of post-max bronchodilator test (Visit 2) (<1hr)
Bronchial Hyperresponsiveness prevalence
Examine how many subjects with PCD have bronchial hyperresponsiveness as evidenced by a decline in FEV1 by 20% from baseline during methacholine challenge test.
Measured: From spirometry baseline measurement (Visit 3) to completion of methacholine challenge test (Visit 3) (<1hr)
Bronchial Hyperresponsiveness prevalence
Examine how many healthy subjects have bronchial hyperresponsiveness as evidenced by an increase in FEV1 of 10% or greater during pre- and post- max bronchodilator test.
Measured: From spirometry baseline measurement (Visit 2) to completion of post-max bronchodilator test (Visit 2) (<1hr)
Bronchial Hyperresponsiveness prevalence comparison
Compare the prevalence of bronchial hyperresponsiveness (examined in Outcome 1, Outcome 2) in PCD subjects to bronchial hyperresponsiveness in age- and gender-matched healthy controls.
Measured: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
Secondary outcomes (2)
Data Collection of PCD genotype/phenotype
These data points will be a compilation of chronic rhinosinusitis, respiratory distress at birth, recurrent upper and lower airway infections, and/or fertility issues, serum biomarkers for atopy, and other clinical features and explore the relationship between PCD and bronchial hyperresponsiveness, which can manifest as coughing, wheezing, and/or chest tightness.
Measured: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
Analysis of the relationship between PCD and atopy
This will address whether bronchial hyperresponsiveness in PCD is due to an underlying predisposition similar to that seen in asthma and whether it could relate to some other mechanism such as antigen mucostasis.
Measured: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Dec 2025
- Study start4 May 2023
- Primary completion30 Apr 2028 (estimated)
- Study completion30 Apr 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Ciliary DyskinesiaStudies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Subjects with Primary Ciliary Dyskinesia and Healthy subjects (controls)
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Evans Machogu, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07288827
- Sponsor's protocol ID
- 16122
- Acronym
- BHR
- Status last verified
- Nov 2025
- First submitted
- 20 Nov 2025
- Last updated
- 17 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center Study Examining Bronchial Hyperresponsiveness in Primary Ciliary Dyskinesia
- ClinicalTrials.gov ID
- NCT07288827
- Acronym / sponsor ID
- BHR / 16122
- Conditions
- Primary Ciliary Dyskinesia, Healthy
- Keywords
- Primary Ciliary Dyskinesia, bronchial hyperresponsiveness
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- 4 May 2023 (actual)
- Primary completion
- 30 Apr 2028 (estimated)
- Study completion
- 30 Apr 2028 (estimated)
- First posted
- 17 Dec 2025 (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 purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness".
Detailed description
The purpose of this study is to look at children with PCD and see if they have another condition called "bronchial hyperresponsiveness". This is when some of the air tubes ("bronchi") are very sensitive ("hyperresponsive") to certain things in the environment such as pollen, mold, pet dander, changes in temperature, and viruses. Children with PCD are often prescribed daily inhaled steroids to help make their air tubes less sensitive, but there are few research studies showing that children with PCD really need this medication.
In this study, the investigators will simulate the above triggers for sensitive air tubes with a medication called methacholine. Checking how fast children can breathe out after giving methacholine is sometimes done when doctors think a child may have asthma, a condition where the breathing tubes are very sensitive. By measuring the participant's breathing before and after giving this medication, the investigators will see whether their air tubes are sensitive, or "hyperresponsive". It is possible that methacholine could make the participants temporarily feel short of breath or start wheezing. To avoid this, the investigators will start by giving a small dose of methacholine and measuring the participants breathing, then give larger amounts of methacholine until the investigators see certain changes in the participants breathing or until the largest dose allowed is reached. If the participant starts to have symptoms such as shortness of breath, chest tightness, or wheezing, the investigators will give them a medicine called albuterol which immediately opens the air tubes and makes the symptoms go away. Any discomfort caused by the methacholine is temporary and there are no long-term effects associated with using it.
If the participants agree to participate in the study, they will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, they will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw.
The investigators will also collect information about the participants from your medical record, including demographic information, medical history, and disease history. If they have completed pulmonary function testing (testing that shows how well lungs are working) in the past, they may only be eligible for Visit 1.
For scheduling convenience, visits 1 and 2 may occur on the same day. The participants will be in this study up to one month if they participate in visits 1 and 2, and up to five months if they are eligible for visit 3.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Subjects with Primary Ciliary Dyskinesia and Healthy subjects (controls)
| Arm | Type | Receives |
|---|---|---|
| Subjects with Primary Ciliary Dyskinesia | Experimental | Procedure: Lung Function Testing, Procedure: Phlebotomy, Procedure: Allergy skin prick testing, Procedure: Methacholine ChallengeSubjects will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, subjects will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw. |
| Healthy Subjects | Experimental | Procedure: Lung Function Testing, Procedure: Phlebotomy, Procedure: Allergy skin prick testing, Procedure: Methacholine ChallengeSubjects will come to Riley Hospital for Children for at least two visits with the opportunity for a third visit. Each visit should take between 2 and 2.5 hours. During the visits, subjects will complete multiple breathing tests, inhale certain medications, complete a family history questionnaire, have skin allergy testing, and do a blood draw. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Confirmed diagnosis of PCD per standard diagnostic criteria4 and positive genetics
- Age greater than or equal to 6 years (no upper age limit)
- Any gender or race
- Able to perform pulmonary function testing (historical documentation of reversibility will be accepted)
Key exclusion criteria (2 listed)
- history of current pneumothorax
- inability to perform pulmonary function testing
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 |
|---|---|---|
| Lung Function TestingAlso: spirometrywill include baseline spirometry (pre- and post- max bronchodilator). All testing will be done according to American Thoracic Society/European Respiratory Society guidelines. Participants will be asked to refrain from taking any asthma medications, including inhaled corticosteroids, short- and long-acting bronchodilators, leukotriene receptor antagonists, and long-acting muscarinic antagonists, for 24 hours prior to any spirometry. This activity will take place at a clinical research center at the respective participating institution. | Procedure or surgery | Healthy Subjects, Subjects with Primary Ciliary Dyskinesia |
| PhlebotomyAlso: blood drawwill be obtained at visit 2. Up to ten (10) ml of blood will be collected for measurement of serum biomarkers of atopy, based on whether participants prefer to receive an allergy skin prick test or have antigen-specific IgE levels tested. In the event a subject refuses phlebotomy, historical results up to one year old may be used in lieu of prospective results. Any remaining blood samples will be banked either for use in future studies or in the event that additional serum biomarkers are added to this study. | Procedure or surgery | Healthy Subjects, Subjects with Primary Ciliary Dyskinesia |
| Allergy skin prick testingmay be completed at visit 2. Subjects will be instructed to withhold first-generation antihistamines for 3 days and second-generation antihistamines for 7 days prior to the test. If patients prefer to have serum antigen-specific IgE levels run with the required serum biomarkers of atopy, then skin prick testing will be omitted. | Procedure or surgery | Healthy Subjects, Subjects with Primary Ciliary Dyskinesia |
| Methacholine ChallengeAlso: MCTIf the subject does not demonstrate a bronchodilator response in FEV1 of 10% or greater, and does not have a historical MCT on file, MCT will be performed. Following inhalation of saline, methacholine (MCh) will be inhaled in quadrupling concentrations starting with 0.0625 mg/ml and continuing until the MCh concentration required for FEV1 to decrease by 20% from baseline (PD20) is achieved or a maximum MCh concentration of 16 mg/ml is inhaled. | Procedure or surgery | Healthy Subjects, Subjects with Primary Ciliary Dyskinesia |
Primary outcome
Bronchial Hyperresponsiveness prevalence
Time frame: From spirometry baseline measurement (Visit 2) to completion of post-max bronchodilator test (Visit 2) (<1hr)
Examine how many subjects with PCD have bronchial hyperresponsiveness as evidenced by an increase in FEV1 of 10% or greater during pre- and post- max bronchodilator test.
Bronchial Hyperresponsiveness prevalence
Time frame: From spirometry baseline measurement (Visit 3) to completion of methacholine challenge test (Visit 3) (<1hr)
Examine how many subjects with PCD have bronchial hyperresponsiveness as evidenced by a decline in FEV1 by 20% from baseline during methacholine challenge test.
Bronchial Hyperresponsiveness prevalence
Time frame: From spirometry baseline measurement (Visit 2) to completion of post-max bronchodilator test (Visit 2) (<1hr)
Examine how many healthy subjects have bronchial hyperresponsiveness as evidenced by an increase in FEV1 of 10% or greater during pre- and post- max bronchodilator test.
Bronchial Hyperresponsiveness prevalence comparison
Time frame: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
Compare the prevalence of bronchial hyperresponsiveness (examined in Outcome 1, Outcome 2) in PCD subjects to bronchial hyperresponsiveness in age- and gender-matched healthy controls.
2 secondary outcomes
Data Collection of PCD genotype/phenotype
Time frame: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
Analysis of the relationship between PCD and atopy
Time frame: From first Visit 2 baseline measurement of first subject enrolled to either: last Visit 3 visit of PCD subject who may complete Visit 3; or last Visit 2 visit of PCD subject or Healthy subject, whichever comes latest in enrollment (up to five years)
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 17 Dec 2025.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Lung Function Testing, Phlebotomy and Allergy skin prick testing in people with Primary Ciliary Dyskinesia and Healthy, run by Indiana University at 1 site in United States.
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 2 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: looking for participants now, as of the sponsor's last update on 17 Dec 2025. 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 United States. 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 Fanmuyi Yang, PhD (317-274-8895, fy5@iu.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bronchial Hyperresponsiveness prevalence (time frame: From spirometry baseline measurement (Visit 2) to completion of post-max bronchodilator test (Visit 2) (<1hr)). 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?
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 17 Dec 2025.