NCT03652090

Brief Summary

characterization of CFTR function and expression in nasal primary cells collected from patients with cystic fibrosis in comparison to their parents, healthy heterozygotes and healthy controls

Trial Health

100
On Track

Trial Health Score

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

Enrollment
112

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Sep 2010

Longer than P75 for all trials

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

Study Start

First participant enrolled

September 1, 2010

Completed
5.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 3, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 3, 2016

Completed
2.5 years until next milestone

First Submitted

Initial submission to the registry

August 28, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 29, 2018

Completed
Last Updated

March 12, 2019

Status Verified

March 1, 2019

Enrollment Period

5.5 years

First QC Date

August 28, 2018

Last Update Submit

March 8, 2019

Conditions

Cystic Fibrosis

Keywords

biomarker

Outcome Measures

Primary Outcomes (1)

  • variation in the short-circuit-current (Isc) after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770)

    The short-circuit-current (Isc) was measured under voltage clamp conditions. Inhibitors and activators were added after stabilization of baseline Isc. The sum of the change after Forskolin (Forskolin)/IBMx and VX-770 (∆IscFsk/IBMx+VX-770) served as an index of CFTR function.

    1 day

Secondary Outcomes (1)

  • percentage of cells displaying apical staining

    1 day

Study Arms (3)

cystic fibrosis patients

Cystic fibrosis patients carrying to 2 CFTR mutations undergoing cell sampling

Procedure: cell sampling

healthy heterozygotes

healthy heterozygotes carrying 1 CFTR mutations undergoing cell sampling

Procedure: cell sampling

healthy control

subject with no evidence of any symptoms compatible with Cystic Fibrosis undergoing cell sampling

Procedure: cell sampling

Interventions

cell samplingPROCEDURE

nasal brushing to collect cells

cystic fibrosis patientshealthy controlhealthy heterozygotes

Eligibility Criteria

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

patients with Cystic Fibrosis with 2 mutations in CFTR * healthy heterozygotes with 1 mutation in CFTR * healthy subjects with no familial history of Cystic Fibrosis and no symptoms compatibel with Cystic Fibrosis

You may qualify if:

  • patients with Cystic Fibrosis with 2 mutations in CFTR
  • healthy heterozygotes with 1 mutation in CFTR
  • healthy subjects with no familial history of Cystic Fibrosis and no symptoms suggesting Cystic Fibrosis

You may not qualify if:

  • smoking

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Biospecimen

Retention: NONE RETAINED

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MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

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

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER GOV
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Professor

Study Record Dates

First Submitted

August 28, 2018

First Posted

August 29, 2018

Study Start

September 1, 2010

Primary Completion

March 3, 2016

Study Completion

March 3, 2016

Last Updated

March 12, 2019

Record last verified: 2019-03