Mucus Clearance Therapy for Primary Ciliary Dyskinesia
Trial Summary
What is the purpose of this trial?
The purpose of this study is to measure mucociliary clearance (MCC) in groups of subjects with the disease Primary Ciliary Dyskinesia (PCD) caused by mutations in different genes, and compare to healthy subjects. Some of these genes are associated with a milder clinical phenotype. This study seeks to determine if the milder phenotype is a result of mutations in a set of specific genes. The hypothesis is that subjects with PCD caused by mutations in the milder group will maintain a low, but significant rate of mucociliary clearance, while patients with mutations in genes in the more severe group will have a complete absence of mucociliary clearance. These studies will help inform future treatment strategies.
Will I have to stop taking my current medications?
The trial requires participants to stop taking certain medications that may impact the study results, including steroids, beta antagonists, non-steroidal anti-inflammatory agents, beta-blocking medications, multivitamins, Vitamin C or E, and herbal medications. There is a specific period of 4 days before the treatment visit during which some of these medications should not be taken.
What data supports the effectiveness of the treatment for mucus clearance in Primary Ciliary Dyskinesia?
Is Mucus Clearance Therapy using Tc-99m sulfur colloid safe for humans?
How does the mucus clearance treatment for primary ciliary dyskinesia differ from other treatments?
This treatment is unique because it involves the use of a nebulized epithelial sodium channel (ENaC) blocker, idrevloride, which helps improve lung function by blocking sodium channels to keep mucus hydrated and easier to clear, unlike other treatments that may not target this specific mechanism.134910
Research Team
Lawrence Ostrowski, PhD
Principal Investigator
University of North Carolina at Chapel Hil
Eligibility Criteria
This trial is for adults over 18 with Primary Ciliary Dyskinesia (PCD) due to specific genetic mutations. Participants need a confirmed PCD diagnosis, normal lung function tests, and no other lung diseases like asthma or cystic fibrosis. Women must not be pregnant unless they've had certain surgeries.Inclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Baseline Measurement
Participants undergo baseline mucociliary clearance measurement using radiolabeled Tc99m-sulfur colloid and gamma imaging
Post-Albuterol Measurement
Participants receive Albuterol and undergo further mucociliary clearance measurement
Cough Clearance Measurement
Participants perform cough clearance assessment over a 30-minute period using gamma imaging
Follow-up
Participants are monitored for safety and effectiveness after the study procedures
Treatment Details
Interventions
- Albuterol
- Technetium99m - Sulfur Colloid (Tc99m-SC)
Find a Clinic Near You
Who Is Running the Clinical Trial?
University of North Carolina, Chapel Hill
Lead Sponsor
National Heart, Lung, and Blood Institute (NHLBI)
Collaborator