International registry for patients with Primary Ciliary Dyskinesia
International Prospective Primary Ciliary Dyskinesia (PCD) Registry for Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
University Hospital Muenster · NCT02419365
This study is creating a registry to gather information about people with Primary Ciliary Dyskinesia to help improve treatments and find patients for future research trials.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2000 (estimated) |
| Sex | All |
| Sponsor | University Hospital Muenster (other) |
| Locations | 2 sites (Münster, North Rhine-Westphalia and 1 other locations) |
| Trial ID | NCT02419365 on ClinicalTrials.gov |
What this trial studies
This registry aims to systematically collect and analyze data on the manifestation, progression, and treatment of Primary Ciliary Dyskinesia (PCD) to support epidemiological research. It is part of the European Reference Network for Rare Lung Diseases and follows EU guidelines for interoperability of rare disease registries. The registry allows for data entry through a secure online platform, enabling authorized personnel to contribute and access data relevant to their specific centers. The goal is to identify patient groups suitable for multi-center trials and enhance understanding of PCD.
Who should consider this trial
Good fit: Ideal candidates for this registry include patients of any age with clinical presentations consistent with PCD and specific diagnostic findings.
Not a fit: Patients who do not meet the diagnostic criteria for PCD or are unable to provide informed consent may not benefit from this registry.
Why it matters
Potential benefit: If successful, this registry could improve the understanding and management of Primary Ciliary Dyskinesia, leading to better treatment options for patients.
How similar studies have performed: Other registries for rare diseases have shown success in improving patient outcomes and facilitating research, making this approach promising.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria Patients of any age who fulfil the diagnostic criteria below are eligible: Clinical presentation consistent with PCD and consistent findings specific for PCD in at least two of the following methods: high frequency video microscopic finding transmission electron microscopy finding immunofluorescence finding low nasal NO concentration/production demonstration of biallelic disease-causing mutations by genotyping Given the complexity of diagnosing PCD, it is anticipated that not all patients will meet these definite diagnostic criteria. Therefore, individuals with typical clinical symptoms and only one abnormal diagnostic test are also eligible to enter the registry. These cases usually are considered to have a possible PCD diagnosis with exceptions made on an individual basis. Exclusion Criteria Failure or unwillingness to give written informed consent. Missing qualification to perform legal acts or insufficient cognitive ability to give informed consent. A second disease is no exclusion criteria for including data in the PCD-Registry, especially since it is one of the fundamental goals, to learn about co-morbidities.
Where this trial is running
Münster, North Rhine-Westphalia and 1 other locations
- University Hospital Münster — Münster, North Rhine-Westphalia, Germany (RECRUITING)
- University Hospital Muenster, Department of General Pediatrics — Münster, Germany (RECRUITING)
Study contacts
- Study coordinator: Petra Pennekamp, PhD
- Email: petra.pennekamp@ukmuenster.de
- Phone: +4925183
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Primary Ciliary Dyskinesia