Long Term Significance (Survival) of LCI in Patients With Cystic Fibrosis

NCT01951833 | Withdrawn DMC

• 1 other identifier: LCI-01-St-Luc
• Study Type: observational
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

The current dream in CF research is to discover safe drugs that correct the basic defect and prevent lung disease, allowing patients without significant lung damage to live nearly normal lives with a dramatic increase in life expectancy and without the burden of current treatment. The compound VX-770 (Ivacaftor Ò) is hoped to be the first milestone along this way. Progression of lung disease is now so gradual in many centres that sensitive indicators of early lung disease (small airways disease) are critically needed to assess the effects of such new treatments. In this context, assessment of ventilation inhomogeneity by the measurement called Lung clearance index (LCI) seems to be the most promising tool. However, to get approval by health authorities, new measures used in drug evaluation need to fulfill strict criteria. For LCI, the investigators still need to prove its long term significance: How well does the LCI measurement predict the long term lung disease course? Therefore, in this study the investigators want to measure LCI at baseline in a large patient cohort and establish how well it predicts the patients' disease course over the next 2 years.

Conditions

Cystic Fibrosis (CF)

Keywords

Lung clearance index (LCI)

Outcome Measures

Primary Outcomes (1)

• Lung Clearance Index (Predictive value and change of)

  Evaluation of LCI is repeated at 1 week and 3 months to assess short and intermediate repeatability. LCI and spirometry will be repeated after 22-24 months after the baseline measurement. This will allow to evaluate tracking of LCI as well as FEV1 % predicted in the total patient cohort as well as in the patients with FEV1 above 80% predicted.

  Baseline, 1 week, 3 months and 2 years

Secondary Outcomes (1)

• FEV1 (Predictive value and change of)

  baseline and 2 years

Other Outcomes (1)

• Symptom score

  1 week, 3 months

Study Arms (2)

Cystic fibrosis (Ecomedics vs NDD)

No treatment, observational

Device: EasyOne Pro and Ecomedics

Healthy (Ecomedics vs NDD)

Free of respiratory symptoms for at least two weeks and will not have any chronic or recurrent chest problem. No treatment, observational

Device: EasyOne Pro and Ecomedics

Interventions

EasyOne Pro and Ecomedics DEVICE

LCI will be assessed with 2 different devices : EasyOne Pro and Ecomedics

Cystic fibrosis (Ecomedics vs NDD); Healthy (Ecomedics vs NDD)

Eligibility Criteria

• Age: 6 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with Cystic Fibrosis ( 6 years and older and FEV1 above 40% predicted) will be invited to participate in this prospective non interventional study in 2 CF centers (UCL and UZ Leuven)

You may qualify if:

• FEV1(% predicted) \> 40%

You may not qualify if:

• FEV1(% predicted) \< 40%
• Exacerbation during baseline measurement

Sponsors & Collaborators

• University Hospital St Luc, Brussels: lead
• KU Leuven: collaborator

Study Sites (1)

Cliniques Universitaires Saint-Luc (UCL)

Woluwé-Saint-Lambert, Brussels Capital, 1200, Belgium

Location

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Lebecque Patrick, PhD

  Cliniques universitaires Saint-Luc- Université Catholique de Louvain

  STUDY DIRECTOR

• Poncin William

  Cliniques universitaires Saint-Luc- Université Catholique de Louvain

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physiotherapist

Study Record Dates

• First Submitted: March 14, 2013
• First Posted: September 27, 2013
• Study Start: January 1, 2013
• Primary Completion: May 1, 2015
• Study Completion: May 1, 2017
• Last Updated: January 15, 2015

Record last verified: 2013-11

Locations

BE (1)