Primary Ciliary Dyskinesia (PCD) Bronchiectasis
Conditions
Interventions
Group 1: Inhalation of hypertonic saline (6% NaCl) 4 ml twice daily via an eFlow® rapid nebulizer system with eTrack® Controller for 8 weeks
Group 2: Inhalation of isotonic saline (0.9% NaCl) 4 ml twi
Sponsors
CAPNETZ STIFTUNG
Eligibility
Sex/Gender
All
Age
12 Years to No maximum
Inclusion criteria
Inclusion criteria: - All subjects must have evidence supportive of a PCD diagnosis • ppFEV1 =40 percentage points adjusted for age, sex, and height, according to the Global Lung Function Initiative (GLI) predicted values at screening • LCI =7.5 at screening • Stable concomitant respiratory therapy according to local or national standards (Raidt et al 2020) for last 4 weeks prior to screening • Ability to comply with study interventions, study visits and study procedures as defined by the protocol
Exclusion criteria
Exclusion criteria: • Diagnosis of cystic fibrosis (CF), including at least 1 of the following: Documented sweat chloride concentration =60 mmol/l by quantitative pilocarpine iontophoresis; Abnormal nasal potential difference (NPD) or intestinal current measurement (ICM) test; 2 CF-causing mutations in the CFTR gene; Subjects with only 1 mutation in the CFTR gene and a sweat chloride concentration =60 mmol/l by quantitative pilocarpine iontophoresis. • History of any organ transplantation. • Significant congenital heart defects, other than a laterality defect, at the discretion of the investigator. • Diagnosis of bronchiectasis due to another condition (e.g. CF) • Pulmonary exacerbation in the last 2 weeks before screening or within the run-in phase, defined as onset of =3 new or worsening symptoms of (Lucas et al 2019) • Claustrophobia (only MRI sub-study) • Other conditions/situations which represent a contraindication for conducting MRI (only MRI sub-study) • Female patient currently being pregnant and/or nursing • Any disease or condition that, in the opinion of the investigator, interferes with the ability to safely complete the study or adhere to the study requirements, including substance abuse • SpO2 <90% at rest and room-air at screening • Planned surgery during the study period • History of clinically significant bronchoconstriction after inhalation of nebulized hypertonic saline (defined by emergency symptoms or an absolute FEV1 decline of =20 percent points 30-60 min after hypertonic saline application) or clinically significant hemoptysis in year prior to screening • Known other intolerance to the experimental treatment (hypertonic saline, e.g. MucoClear® 6%) • History of at least 2 sputum or throat swab cultures yielding Burkholderia cepacia complex or Mycobacterium abscessus complex or Mycobacterium avium intracellulare complex within the previous 2 years • Previous participation in other interventional study within 28 days prior to visit V1 (end of run-in / start of treatment) or during the current study (note: participation in a past or concurrent observational study is acceptable).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Change in lung clearance index (LCI) after 8 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Change in lung function parameters FEV1, ppFEV1, FVC, ppFVC, FEF25-75, ppFEF25-75 2) Change in age-specific QOL: QOL-PCD, mMRC, EQ-5D-5L 3) Change in distance of 6-minute walk test (in meters) | — |
Countries
Germany
Contacts
Public ContactIsabell Pink
Medizinische Hochschule Hannover
Outcome results
None listed