Thermal spa treatment to improve symptoms of primary ciliary dyskinesia

Interest of Thermal Spa Treatment in Improving ENT-Related Quality of Life in Patients With Primary Ciliary Dyskinesia

We will try a 3-week thermal spa treatment in adults with primary ciliary dyskinesia to see if it improves ear, nose, and throat symptoms, lowers infection and antibiotic rates, and helps hearing and lung function.

Quick facts

• Phase: Not applicable
• Study type: Interventional
• Enrollment: 35 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Association Francaise pour la Recherche Thermale (Academic / other)
• Locations: 1 site (Nice)
• Trial ID: NCT07029594 on ClinicalTrials.gov

What this trial studies

Adults with confirmed primary ciliary dyskinesia are randomized to either start with a 3-week balneotherapy (thermal spa) course followed by 6 months of follow-up, or to receive standard care for 6 months and then the spa treatment with further follow-up. The trial collects ENT and lung function measures, quality-of-life questionnaires, hearing tests, and nasal swabs, and tracks infection frequency and antibiotic use. Primary comparisons focus on ENT symptom change and daily quality of life, with secondary outcomes including hearing and pulmonary function. The design allows within- and between-group comparisons over a 12-month period due to the sequential crossover for the delayed group.

Who should consider this trial

Good fit: Adults over 18 with a confirmed PCD diagnosis (by electron microscopy and/or genetic testing), stable maintenance treatment for at least one month, performance status ≤1, and no recent thermal spa therapy are ideal candidates.

Not a fit: Patients with severe or unstable comorbidities, recent contraindications to spa therapy (recent DVT, uncontrolled heart failure, labile hypertension, recent unhealed fractures or skin lesions), children, or those unable to travel for the 3-week treatment are unlikely to benefit or be eligible.

Why it matters

Potential benefit: If effective, the thermal spa treatment could reduce ENT symptoms, decrease infection episodes and antibiotic use, and improve hearing, lung function, and overall quality of life for adults with PCD.

How similar studies have performed: Spa/balneotherapy has limited and mixed evidence in chronic ENT and respiratory disorders and has not been well studied specifically in PCD, so this application is relatively novel.

Eligibility criteria

Inclusion Criteria:

* Confirmed diagnosis of Primary Ciliary Dyskinesia (PCD) based on electron microscopy analysis of ciliary ultrastructure and/or genetic testing (i.e., two unambiguous mutations in genes known to be involved in PCD, particularly in cases with normal ciliary ultrastructure \[\~20% of patients\], atypical ultrastructural findings, or suggestive symptoms in a family with a known causal gene).
* Age over 18 years.
* Stable maintenance treatment for at least 1 month prior to inclusion.
* Performance Status (PS) ≤ 1.
* Patient has received the study information sheet and signed the informed consent form.
* Patient has valid health insurance coverage.

Exclusion Criteria:

* Participation in a thermal spa treatment within the past year.
* Severe general health deterioration or debilitating conditions.
* Severe or recent heart failure.
* Labile hypertension.
* Venous insufficiency or deep vein thrombosis within the past 3 months.
* Recent unhealed fracture.
* Unhealed skin lesions (wounds, pressure sores, eczema).
* Contagious or progressive diseases.
* Recent cancer (less than 5 years).
* Ongoing or recent (within the past month) pulmonary or ENT exacerbation at inclusion.
* Ongoing treatment with immunomodulatory or immunosuppressive drugs (due to the risk of potentially severe opportunistic infections).
* Ongoing or recent (within the past month) extra-respiratory or ENT infectious flare-up.
* Positive pre-cure bacteriological testing for multidrug-resistant Pseudomonas aeruginosa, MRSA, Mycobacterium abscessus, or other multidrug-resistant pathogenic bacteria.
* Fever \> 38.5°C.
* Recently worsened dyspnea (\< 1 month).
* History of serious adverse effects or intolerance during a previous thermal spa treatment.
* Participation in another clinical trial with an exclusion period.
* Considered a vulnerable person, as defined by Articles L1121-5 to L1121-8 of the French Public Health Code:

Pregnant women, women in labor, and breastfeeding mothers.

Individuals deprived of liberty by judicial or administrative decision, or those hospitalized without consent under Articles L.3212-1 and L.3213-1 (excluding Article L.1121-8), and persons admitted to a healthcare or social institution for reasons other than research.

Adults under legal protection or unable to give informed consent.

Where this trial is running

Nice

• Institut Universitaire de la Face et du Cou — Nice, France (Recruiting)

Study contacts

• Study coordinator: Colin DEBAIGT

Conditions Primary Ciliary Dyskinesia