Primary Ciliary Dyskinesia Clinical Trial in Edmonton Alberta
NCT03271840
| Observational patient registry
This study has recruited 12 Participants
Primary Ciliary Dyskinesia (PCD) is a rare disease, which means that any single PCD center
has experience with a limited number of patients. PCD Registry is the collection of data
about PCD from many centers and countries who treat children with PCD. Collecting data about
PCD increase the knowledge on PCD, better describe the course of the disease, and help to
better understand the progression of the disease and be used to develop new treatments.
In the PCD registry of Alberta, important information about PCD such as time of diagnosis,
symptoms, and tests which led to the diagnosis, state of health at diagnosis, the progression
of lung function, the occurrence of severe infections, tests and treatments data will be
collected from the patients' medical records.
Details for the study
Population
Patients with PCD
Brief Title
Registry for Primary Ciliary Dyskinesia
Official Title
Registry for Primary Ciliary Dyskinesia: : Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease
Brief Summary
Primary Ciliary Dyskinesia (PCD) is a rare disease, which means that any single PCD center
<br /> has experience with a limited number of patients. PCD Registry is the collection of data
<br /> about PCD from many centers and countries who treat children with PCD. Collecting data about
<br /> PCD increase the knowledge on PCD, better describe the course of the disease, and help to
<br /> better understand the progression of the disease and be used to develop new treatments.
<br />
<br /> In the PCD registry of Alberta, important information about PCD such as time of diagnosis,
<br /> symptoms, and tests which led to the diagnosis, state of health at diagnosis, the progression
<br /> of lung function, the occurrence of severe infections, tests and treatments data will be
<br /> collected from the patients' medical records.
Detailed Description
In order to better characterize the clinical course, and improve the diagnosis and treatment
of PCD, the investigators propose to establish a provincial PCD registry in Alberta. This
registry will be securely linked to the currently existing International Registry for PCD and
will allow more researchers to use the information to do research on PCD.
The investigators will collect demographic data: current age, sex, age, diagnostic findings
and symptoms at diagnosis; in addition, clinical data about PCD which are recorded during the
past and future clinical visits and encounters (e.g. lung function, weight, height,
infections, treatments, and complications) will also be collected.
Outcome Measures
Outcome measures are the tests that investigators perform to prove whether or not a treatment being tested in a clinical trial is having any effect.
Primary
Lung Function
% predicted for age
Primary
Age of diagnosis
Years
Study Criteria
Inclusion Criteria:
- Clinical diagnosis of PCD
- Abnormal findings in at least two of the following tests:
High-frequency video microscopic finding, transmission electron microscopy finding,
immunofluorescence finding, low nasal NO concentration/production, demonstration of
biallelic disease-causing mutations by genotyping.
Exclusion Criteria:
• Failure or unwillingness to give written informed consent.