NCT04395820

Brief Summary

Cystic fibrosis (CF) is the most common lethal inherited disease in Caucasian populations. To improve survival, it is essential to understand the development, progression and treatment of CF lung disease throughout early childhood. Therefore the overall objective is to prospectively assess the clinical utility of novel and non-invasive measuring methods, namely Multiple Breath Washout and functional lung MRI in the longitudinal clinical surveillance of patients with CF and compare the results to those of healthy controls.

Trial Health

75
On Track

Trial Health Score

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

Enrollment
250

participants targeted

Target at P75+ for all trials

Timeline
908mo left

Started Jul 2020

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
active not recruiting

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

Study Progress7%
Jul 2020Dec 2100

First Submitted

Initial submission to the registry

May 5, 2020

Completed
15 days until next milestone

First Posted

Study publicly available on registry

May 20, 2020

Completed
1 month until next milestone

Study Start

First participant enrolled

July 1, 2020

Completed
30.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2050

Expected
50 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2100

Last Updated

November 4, 2020

Status Verified

November 1, 2020

Enrollment Period

30.4 years

First QC Date

May 5, 2020

Last Update Submit

November 3, 2020

Conditions

Cystic FibrosisHealthy

Keywords

Cystic FibrosisMBWMRIAirway growthAirway developmentHealthy

Outcome Measures

Primary Outcomes (2)

  • Multiple Breath Washout

    Longitudinal assessment of lung volume and ventilation inhomogeneity

    Every third month up to 50 years. Healthy controls only during 1 year.

  • Functional MP-MRI

    Longitudinal assessment of percentage of the lung volume with impaired fractional ventilation and relative perfusion

    Every twelfth month up to 50 years. Healthy controls only during 1 year (2 time points).

Secondary Outcomes (12)

  • Morphological MRI

    Every twelfth month up to 50 years. Healthy controls only during 1 year (2 time points)

  • Spirometry: FEV1

    Every third month up to 50 years. Healthy controls only during 1 year.

  • Spirometry: FVC

    Every third month up to 50 years. Healthy controls only during 1 year.

  • Spirometry: FEF

    Every third month up to 50 years. Healthy controls only during 1 year.

  • Body plethysmography: sRAW

    Every third month up to 50 years. Healthy controls only during 1 year.

  • +7 more secondary outcomes

Study Arms (2)

Cystic Fibrosis

Diagnostic Test: Lung function testDiagnostic Test: Imaging

Healthy

Diagnostic Test: Lung function testDiagnostic Test: Imaging

Interventions

Lung function testDIAGNOSTIC_TEST

MBW

Cystic FibrosisHealthy
ImagingDIAGNOSTIC_TEST

MP-MRI

Cystic FibrosisHealthy

Eligibility Criteria

Age3 Years - 18 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Children and adults with CF and healthy volunteers from the local community in Bern and surrounding areas.

You may qualify if:

  • Individuals with CF:
  • Diagnosis of CF
  • Signed written informed consent
  • ≥3 - 18 years of age, depending on the cooperation and if lung function measurements are possible
  • Healthy volunteers:
  • Signed written informed consent
  • Informed consent of participant and if under 18 years, legal representative respectively
  • Children and adults with no history of chronic lung disease or acute respiratory infection in the four weeks prior to the study visit
  • ≥3 - 18 years of age, depending on the cooperation and if lung function measurements are possible

You may not qualify if:

  • Women who are pregnant or breast feeding.
  • Intention to become pregnant during the course of the study
  • Lack of safe contraception, defined as: Female participants of childbearing potential, not using and not willing to continue using a medically reliable method of contraception for the entire study duration, such as oral, injectable, or implantable contraceptives, or intrauterine contraceptive devices, or who are not using any other method considered sufficiently reliable by the investigator in individual cases.
  • Please note that female participants who are surgically sterilised/hysterectomised or post-menopausal for longer than 2 years are not considered as being of child bearing potential.
  • Other clinically significant concomitant disease states (e.g. renal failure, hepatic dysfunction, cardiovascular disease, etc.)
  • Known or suspected non-compliance, drug or alcohol abuse
  • Continuous glucose monitor
  • Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, etc. of the participant
  • Metal in body, e.g. pacemaker
  • Participation in another study with investigational drug within the 30 days preceding and during the present study
  • Subjects which are respiratory insufficient to attend on the lung function measurements (oxygen demand)
  • Subjects who are unable to perform the MRI without sedation
  • Participants which were born preterm (\<36. week of pregnancy)
  • Current smokers
  • In addition for individuals with CF:
  • +3 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Children's Hospital Bern

Bern, 3010, Switzerland

Location

Related Publications (2)

  • O'Sullivan BP, Freedman SD. Cystic fibrosis. Lancet. 2009 May 30;373(9678):1891-904. doi: 10.1016/S0140-6736(09)60327-5. Epub 2009 May 4.

    PMID: 19403164BACKGROUND

  • Ramsey KA, Ranganathan S, Park J, Skoric B, Adams AM, Simpson SJ, Robins-Browne RM, Franklin PJ, de Klerk NH, Sly PD, Stick SM, Hall GL; AREST CF. Early respiratory infection is associated with reduced spirometry in children with cystic fibrosis. Am J Respir Crit Care Med. 2014 Nov 15;190(10):1111-6. doi: 10.1164/rccm.201407-1277OC.

    PMID: 25321321BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

Oropharyngeal swabs

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

Respiratory Function TestsDiagnostic Imaging

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Diagnostic Techniques, Respiratory SystemDiagnostic Techniques and ProceduresDiagnosis

Study Officials

  • Kathryn Ramsey, PhD

    University Children's Hospital Bern

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 5, 2020

First Posted

May 20, 2020

Study Start

July 1, 2020

Primary Completion (Estimated)

December 1, 2050

Study Completion (Estimated)

December 1, 2100

Last Updated

November 4, 2020

Record last verified: 2020-11

Locations

CH(1)