Response to CFTR Modulators in CF Patients Under 18 Years (MODUL-CF)

March 19, 2021 updated by: Isabelle SERMET-GAUDELUS, Societe Francaise de la Mucoviscidose

Evaluation of the Response to CFTR Modulators in Patients With Cystic Fibrosis Less Than 18 Years of Age

CFTR modulators should improve the prognosis of Cystic Fibrosis. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity is an important medical objective given the potential side effects and the high cost of these molecules.

This observational follow-up cohort study is carried out as part of routine care.

The main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy.

The secondary objectives are to evaluate structural impairment at low dose scan at 3 years and 5 years of CFTR modulator treatment, the evolution of respiratory functional parameters, growth, puberty, lung infection, sweat test, quality of life and pancreatic function, as well as tolerance of modulators including liver toxicity.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

Cystic fibrosis (CF) is a deadly disease. This is due to overinfected chronic obstructive pulmonary disease that progresses to end-stage respiratory failure. CFTR modulators should improve the prognosis of CF, as they may slow the progression of patients' lung disease. Assessing their impact in the paediatric population is becoming a major issue. Children and adolescents under the age of 18 are a target cohort because they have a lung disease that is still poorly developed. Early prescription of CFTR modulators is therefore a priority but requires evidence of absence of toxicity. Identifying patients under the age of 18 responding to CFTR modulators as well as detecting possible toxicity, is an important medical objective given the potential side effects and the high cost of these molecules.

The outcomes previously used in Phase III studies (FEV1, frequency of exacerbations, nutritional status) are insufficiently sensitive in this population.

Other criteria need to be analyzed to identify the response to CFTR modulators in the short and medium term. The investigators hypothesize that the assessment of pulmonary structural impairment by low-dose lung CT-scan as part of routine care could be a much more sensitive criterion for the development of lung disease under CFTR modulators.

This observational follow-up cohort study is carried out as part of routine care. It does not involve a specific collection for research. Excess bronchial secretions and blood will be kept instead of being discarded in the event of a possible requalification for research.

The main objective is to assess the evolution of pulmonary structural impairment by low-dose CF scan at the end of the first year of CFTR modulator therapy The secondary objectives are to assess following criteria

  • Tolerance of modulators in this age group, including screening for bronchial reactivity at treatment, early liver toxicity
  • Longitudinal evolution of pulmonary structural impairment by low dose scan at 3 years and 5 years of CFTR modulator treatment
  • Evolution of respiratory functional parameters
  • Measurement by spirometry and plethysmography
  • Lung clearance index (if possible)
  • Longitudinal evolution of bacterial colonization, compared to the year prior to modulating treatment
  • Exacerbations: number, duration, days of antibiotics, hospitalizations, return to stable condition
  • Colonization of bronchial secretions
  • Changes in quality of life
  • Evolution of the sweat test
  • Longitudinal evaluation of pancreatic function
  • Longitudinal evaluation of growth and puberty compared to the year prior to CFTR modulator
  • Growth speed, and bone age
  • Bone mineralization, body composition (if possible)
  • Pubertal markers from 9 years in girls and 10 years in boys
  • Evaluation of glycemic dysregulation if present
  • Preservation of samples taken as part of routine care (serum, bronchial secretions) for possible research use

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
600

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • France
      • Paris, France, 75015
        • Recruiting
        • Sermet-Gaudelus Isabelle
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

No older than 14 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Children with cystic fibrosis under the age of 18 eligible for CFTR modulator thérapies according to french national recommandations

Description

Inclusion Criteria:

Children with cystic fibrosis under the age of 18 under CFTR modulator therapy

Exclusion Criteria:

  • Patients with cystic fibrosis without indication for CFTR modulator therapy
  • Patients over the age of 18
  • Pregnant or lactating women

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
CF children treated with CFTR modul
Cystic fibrosis patients under 18 years treated with CFTR modulators according to french health recommendations observational cohort study
Drug: CFTR Modulators
CFTR modulators as recommended in routine care by the french health authorities
Other Names:
  • Orkambi, Kalydeko...

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Lung Imaging
Time Frame: at initiation, as part of national guidelines
Lung structural injury assessed by Low Dose CT, as part of routine care
at initiation, as part of national guidelines
Lung Imaging
Time Frame: at 1 year, as part of national guidelines
Lung structural injury assessed by Low Dose CT, as part of routine care
at 1 year, as part of national guidelines
Lung Imaging
Time Frame: at 3 years, as part of national guidelines
Lung structural injury assessed by Low Dose CT, as part of routine care
at 3 years, as part of national guidelines
Lung Imaging
Time Frame: at 5 years, as part of national guidelines
Lung structural injury assessed by Low Dose CT, as part of routine care
at 5 years, as part of national guidelines

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
weight in kilogrammes
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
weight in kilogrammes (associated with a retrospective collection in the year prior to treatment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
height in meters
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
height in meters (associated with a retrospective collection in the year prior to treatment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
pubertal evolution
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
pubertal evolution (associated with a retrospective collection in the year prior to treatment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
bronchial infectious exacerbations
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
bronchial infectious exacerbations (associated with a retrospective collection in the year prior to treatment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Expiratory Volume in 1 second(FEV1)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Expiratory Volume in 1 second(FEV1) in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Vital Capacity (FVC)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Vital Capacity (FVC) in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Force Expiratory Flow 50 (FEV50)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Force Expiratory Flow 50 (FEV50) in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Expiratory Flow 25-75 (FEV25-75)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Forced Expiratory Flow 25-75 (FEV25-75) in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Residual Volume (RV)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Residual Volume (RV) in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Total Pulmonary Capacity
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Total Pulmonary Capacity in liter
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Lung Clearance Index - Lung Clearance Index
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Lung clearance of nitrogen
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
colonization of bronchial secretions
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
bacteria, fungi, mycobacteria
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
quality of life questionnaire
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
CFQ questionnaire for children above 8 years: worse 0, better 100
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
ENT quality of life questionnaire
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
SN-score: better score 1, worse 7
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Abdominal quality of life questionnaire
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
better score: 0, worse: 25
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
liver ultrasound
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
liver ultrasound
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
elastometry (data available in centers with the necessary equipment)
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
elastometry (data available in centers with the necessary equipment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
sweat test
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
sweat collection
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
serum and fecal pancreatic biological markers
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
immunoreactive trypsin, lipase, amylase, vitamin A and E, Prothrombin time, and fecal (fecal elastase
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
bone biological markers
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
25OHvitD, Ca, P, PTH, Osteocalcin, IgF1, IgF1BP3, CTX
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
bone maturation
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
Zscore (in relation to height and sex and weight) (data available in centers with the necessary equipment)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
puberty
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
serum dosage of FSH, LH, Estradiol, testosterone Pelvic ultrasound if puberty initiated in girls
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
intestine inflammation
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
fecal Calprotectine
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
glycemic regulation
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
monitoring of glycemic dysregulation ( as routinely done in the centers)
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
side effects: declarative collection and monitoring
Time Frame: longitudinal monitoring of assessments carried out as part of routine care during 5 yrs
declarative collection and monitoring
longitudinal monitoring of assessments carried out as part of routine care during 5 yrs

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Societe Francaise de la Mucoviscidose

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Isabelle Sermet-Gaudelus, MD PhD, Societe Francaise de la Mucoviscidose

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2020

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 1, 2025

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
January 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 21, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 5, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 10, 2020

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 22, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 19, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 0011928

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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