Trials Sponsors Conditions Drugs Pricing

Conditions

Kartagener Syndrome Primary Ciliary Dyskinesia

Outcome Measures

Primary Outcomes (3)

Mean FEV1 Percent Predicted Values in Well State and Sick State
Forced expired volume in 1 second (FEV1) will be assessed by percentage of the predicted value (0-100%).
12 months
Mean Primary Ciliary Dyskinesia-Quality of Life Score in Well State and Sick State
Domains (scales) include physical, emotional, social, school and role functioning; treatment burden; ears and hearing; upper and lower respiratory symptoms; and vitality. The recall period is one week and responses are rated on a 4-point Likert scale.
12 months
Mean PCD-Respiratory Symptom Diary Score Well State and Sick State
The PCD-RSD contains 17 items, 10 on symptoms and 7 on social/emotional impact. The recall period is 24 hours and 15 questions are rated on a 5-point Likert Scale, while two questions are binary (Range: 0-62, 0 being the best and 62 being the worst).
12 months

Study Arms (2)

Primary Ciliary Dyskinesia (PCD) - Well State

Subjects with confirmed PCD in Well State

Primary Ciliary Dyskinesia (PCD) - Sick State

Subjects with confirmed PCD in Sick State

Eligibility Criteria

Age6 Years+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

Subjects diagnosed with PCD

You may qualify if:

Diagnosis of PCD

Sponsors & Collaborators

Stanford University
Othercollaborator
University of North Carolina, Chapel Hill
Otherlead
Children's Hospital Medical Center, Cincinnati

Study Sites (7)

Stanford University

Palo Alto, California, 94304, United States

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Washington University in St. Louis

St Louis, Missouri, 63130, United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Seattle Children's Hospital

Seattle, Washington, 98105, United States

The Hospital for Sick Children

Toronto, Ontario, M5G 0A4, Canada

McGill University

Montreal, Quebec, H4A 3J1, Canada

Biospecimen

Retention: SAMPLES WITHOUT DNA

sputum for inflammatory markers

MeSH Terms

Conditions

Ciliary Motility DisordersKartagener Syndrome

Condition Hierarchy (Ancestors)

Respiratory Tract DiseasesOtorhinolaryngologic DiseasesCiliopathiesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornBronchiectasisBronchial DiseasesRespiratory System AbnormalitiesDextrocardiaHeart Defects, Congenital

Study Officials

Margaret Rosenfeld, MD
Seattle Children's Hospital
PRINCIPAL INVESTIGATOR
Scott Sagel, MD, PhD
Children's Hospital Colorado
PRINCIPAL INVESTIGATOR

Enrollment

105 participants (actual enrollment reported by the registry).

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: PROSPECTIVE
Sponsor Type: OTHER
Responsible Party: SPONSOR

Study Record Dates

First Submitted

November 29, 2021

First Posted

December 17, 2021

Study Start

March 29, 2022

Actual (reported)

Longitudinal Characterization of Respiratory Tract Exacerbations and Treatment Responses in Primary Ciliary Dyskinesia

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Study Arms (2)

Primary Ciliary Dyskinesia (PCD) - Well State

Primary Ciliary Dyskinesia (PCD) - Sick State

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (7)

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Study Arms (2)

Primary Ciliary Dyskinesia (PCD) - Well State

Primary Ciliary Dyskinesia (PCD) - Sick State

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (7)

Biospecimen

MeSH Terms

Conditions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations