Registry for Primary Ciliary Dyskinesia

Sponsor

University of Alberta (Other)

Overall Status

Recruiting

CT.gov ID

NCT03271840

Collaborator

(none)

Enrollment

Location

160.2

Anticipated Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Primary Ciliary Dyskinesia

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.

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

30 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

Registry for Primary Ciliary Dyskinesia: : Systematic Data Collection on Incidence, Clinical Presentation, Treatment and Course of the Disease

Actual Study Start Date :

Aug 26, 2017

Anticipated Primary Completion Date :

Jan 1, 2030

Anticipated Study Completion Date :

Dec 31, 2030

Outcome Measures

Primary Outcome Measures

Age of diagnosis [2017-2030]
Years
Lung Function [2017-2030]
% predicted for age

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Alberta	Edmonton	Alberta	Canada	T6G2C6

Sponsors and Collaborators

University of Alberta

Investigators

Principal Investigator: Israel Amirav, MD, University of Alberta

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

University of Alberta

ClinicalTrials.gov Identifier:

NCT03271840

Other Study ID Numbers:

pro00074669

First Posted:

Sep 5, 2017

Last Update Posted:

May 20, 2022

Last Verified:

May 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Ciliary Motility Disorders

Dyskinesias

Study Results

No Results Posted as of May 20, 2022