Clearance in Primary Ciliary Dyskinesia

NCT07699302 | Not Yet Recruiting

• 1 other identifier: 25-1650
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief Summary

Understanding mechanisms of mucus clearance from the airways may lead to treatments in muco-obstructive lung conditions. Mucociliary clearance (MCC) scans are a way to measure the rate at which a person's lungs can clear inhaled particles. Through a single-arm pilot study in participants with primary ciliary dyskinesia (PCD), the investigators will evaluate the effect of exercise as a way to increase clearance through a mechanism called gas liquid transport (GLT) where the faster flow of air through the lungs during exercise is able to clear more particles. The investigators will repeat MCC measures with exercise after administering hypertonic saline (HS) to evaluate the effects of mucus hydration on both GLT and cough clearance (CC). Aim 1. To quantify non-ciliary, non-cough mechanisms of mucus clearance (i.e., GLT) in PCD through an MCC protocol that introduces a standardized exercise intervention after isotope delivery. Aim 2. To evaluate whether exercise-induced GLT and/or CC are responsive to hydrator intervention, using a short acting bronchodilator with 7% HS prior to isotope delivery followed by MCC with exercise intervention.

Conditions

Primary Ciliary Dyskinesia; PCD

Keywords

exercise; primary ciliary dyskinesia; airway clearance; mucociliary clearance; hypertonic saline; cough clearance; gas liquid transport

Outcome Measures

Primary Outcomes (3)

• Whole Lung Total GLT

  Through MCC scan with built in exercise protocol, the investigators will measure the effects of exercise on rates of clearance of radiolabeled isotopes from the lungs, thought to represent GLT. The investigators will measure the difference between retained particles from immediately before (after collection of 16 minutes of baseline MCC data through eight 2-minute images) and immediately after 20 minutes of moderate intensity exercise on a cycle ergometer (40 minutes after start of MCC data collection). This will represent Active GLT. The investigators will then resume measurement of MCC clearance rate by serial 2-minute image collection of the whole lung from 40 minutes to 70 minutes. This will represent Sustained GLT. The sum of Active and Sustained GLT will represent Whole Lung GLT. This measurement will occur at either Visit 2 or Visit 3, depending on visit sequence randomization.

  Either Day 1 (Visit 2) or up to Day 28 (Visit 3) depending on randomization to sequence intervention

• Change in Whole Lung Total GLT after HS pretreatment

  After pretreatment with levalbuterol and 7% HS, participants will undergo an MCC scan with built in exercise protocol. The investigators will measure the effects of exercise on rates of clearance of radiolabeled isotopes from the lungs, thought to represent GLT. The investigators will measure Active GLT and Sustained GLT after HS pretreatment, which taken together will represent Whole Lung GLT after HS pretreatment. This measurement will occur at either Visit 2 or Visit 3 depending on visit sequence randomization. The difference between Whole Lung GLT from exercise alone will be compared with the HS pretreatment Whole Lung GLT.

  Day 3 to 28, depending on timing of Visit 3

• Change in Cough Clearance (CC) after HS Pretreatment

  The investigators will measure the effects of exercise and HS pretreatment on rates of clearance of radiolabeled isotopes from the lungs during a period of controlled coughs at regular intervals. During Visit 2 and Visit 3 MCC scans, from minutes 70 through 100 of MCC data collection, participants will be instructed to perform 30 voluntary huff-cough maneuvers while serial 2-minute images are obtained. The change in clearance during this time will represent CC. At one of these visits (either Visit 2 or Visit 3 depending on randomization to intervention sequence), participants will undergo pretreatment with levalbuterol and 7% HS prior to the MCC scan with exercise protocol. The difference between CC after exercise alone and CC after exercise with HS pretreatment will be measured.

  Day 3 to 28, depending on timing of Visit 3

Study Arms (1)

Exercise with and without HS pretreatment

EXPERIMENTAL

All participants will undergo interventions of exercise alone, and exercise with HS pretreatment, in two separate study visits according to randomization to intervention sequence.

Behavioral: Exercise; Other: HS pretreatment with Exercise

Interventions

Exercise BEHAVIORAL

At either V2 or V3, after 16 minutes of baseline MCC data collection, participants will perform 20 minutes of moderate-intensity exercise based on the Borg Rating of Perceived Exertion (RPE) scale, on a cycle ergometer. The goal through this moderate intensity exercise is to increase respiratory rate and airflow velocity, thereby potentially inducing GLT. Participants will then resume MCC scan data collection post-exercise to continue to measure the effects induced by exercise on clearance from the lungs.

Exercise with and without HS pretreatment

HS pretreatment with Exercise OTHER

At either V2 or V3, prior to the start of baseline MCC scanning, participants will undergo pretreatment with 4 puffs of inhaled levalbuterol (90 mcg per actuation, via spacer) followed by 4 ml of 7% HS via nebulizer. Participants will then start MCC scan data collection, and after 16 minutes of baseline MCC collection they will then complete 20 minutes of moderate intensity exercise according to the Borg RPE on a cycle ergometer. After 20 minutes, MCC scan will resume to continue to measure rates of clearance following HS pretreatment and exercise.

Exercise with and without HS pretreatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• years old or older
• Diagnosis of PCD with supporting genetics
• Lung function with forced expiratory volume in 1 second (FEV1) percent predicted \>30%
• Ability to exercise for 20 minutes (Exercise Assessment performed at screening visit)

You may not qualify if:

• Intolerance to 7% HS
• Failure of HS tolerance test (HSTT) at visit 1 (V1), defined as a decline of 10% in FEV1 from baseline pre-bronchodilator (BD) FEV1 after 7% HS
• Intolerance to levalbuterol
• Exercise-induced asthma
• Complex congenital heart disease or other exercise limitation
• Lung transplantation or listed for lung transplantation
• History of lung resection of the lung analyzed by MCC (depending on situs status)
• Pregnancy or unwillingness to use approved birth control during study period
• Recent hemoptysis (\>60 ml in the last 3 months)
• Pulmonary exacerbation in preceding 4 weeks
• Change in pulmonary therapies within preceding 4 weeks
• Resting or exertional O2 requirement (nocturnal acceptable)
• Tobacco / smoking history (any within last year, and \>10 pack year history in past)
• More than 2 chest computed tomography (CT) scans in the past year or a combination of procedures believed to have exposed the lungs to \>150 millisieverts (mSv), including from other research studies
• Participation in another interventional trial within 30 days

Sponsors & Collaborators

• Cystic Fibrosis Foundation: collaborator
• University of North Carolina, Chapel Hill: lead

Study Sites (1)

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27278, United States

Location

MeSH Terms

Conditions

Ciliary Motility Disorders; Motor Activity

Interventions

Exercise

Condition Hierarchy (Ancestors)

Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Ciliopathies; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Behavior

Intervention Hierarchy (Ancestors)

Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Officials

• Katherine A Despotes, MD

  University of North Carolina, Chapel Hill

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Katherine A Despotes, MD

CONTACT

919-966-1055; katherine.despotes@unchealth.unc.edu

Corinne Taylor

CONTACT

corinne.lawler@unc.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: This is a single arm pilot study in PCD, where all patients will undergo the interventions of exercise and exercise after pretreatment with HS and levalbuterol as part of MCC scans during study visits. At the screening visit (V1), the investigators will randomize participants to the sequence which they complete the interventions/study visits (exercise alone first vs exercise with HS pretreatment first at V2). Participants will then subsequently complete the other type of study visit at V3.

Study Record Dates

• First Submitted: June 29, 2026
• First Posted: July 13, 2026
• Study Start: July 30, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2027
• Last Updated: July 13, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)