NCT03271840

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Aug 2017

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 26, 2017

Completed
Same day until next milestone

Study Start

First participant enrolled

August 26, 2017

Completed
10 days until next milestone

First Posted

Study publicly available on registry

September 5, 2017

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 25, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 25, 2021

Completed
Last Updated

June 6, 2023

Status Verified

June 1, 2023

Enrollment Period

4.3 years

First QC Date

August 26, 2017

Last Update Submit

June 5, 2023

Conditions

Primary Ciliary Dyskinesia

Outcome Measures

Primary Outcomes (2)

  • Age of diagnosis

    Years

    2017-2030

  • Lung Function

    % predicted for age

    2017-2030

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with PCD

You may qualify if:

  • 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.

You may not qualify if:

  • Failure or unwillingness to give written informed consent.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Alberta

Edmonton, Alberta, T6G2C6, Canada

Location

MeSH Terms

Conditions

Ciliary Motility Disorders

Condition Hierarchy (Ancestors)

Respiratory Tract DiseasesOtorhinolaryngologic DiseasesCiliopathiesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, Inborn

Study Officials

  • Israel Amirav, MD

    University of Alberta

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
10 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 26, 2017

First Posted

September 5, 2017

Study Start

August 26, 2017

Primary Completion

November 25, 2021

Study Completion

November 25, 2021

Last Updated

June 6, 2023

Record last verified: 2023-06

Locations

CA(1)