An observational study in Cystic Fibrosis, sponsored by Assistance Publique - Hôpitaux de Paris. Completed at 1 site in France. Open to participants aged 12 Years to 20 Years. Per ClinicalTrials.gov, last updated 2025-11-20.
Sponsored by Assistance Publique - Hôpitaux de Paris · Observational
The aim of this project is to evaluate the psychological reshuffle induced by ORKAMBI. The particular focus of this study is the consequence of its introduction on anxiety, depression, quality of life and adherence to all cystic fibrosis (CF) treatment. To answer this question investigators will monitor the psychological function of CF adolescents and young adults treated with ORKAMBI and compare them to CF adolescents and young adults not treated with ORKAMBI.
Introduction of KALYDECO then ORKAMBI, as a personalized medicine in the therapeutic strategy of Cystic Fibrosis (CF) may deeply modify the prognosis of patients with CF. These approaches realize the dream of short-circuiting the molecular mechanism of the disease and therefore opens the perspective of an extended and functional survival. It suddenly offers the possibility of a completely new future to the patient and his family, and therefore must deeply impact on the psychological function of all involved. Moreover, those new therapies may also modify the adherence to CF basic treatment, as it brings a new feeling of good health and the hope of a longer life. For that reason there is a need to investigate modifications of the psychological state of mind induced by the treatment with ORKAMBI in CF adolescents and young adults.
This project aims to understand the psychological impact and to study the consequences of the introduction of ORKAMBI on adherence to CF treatment, anxiety, depression and quality of life.
To answer this question, investigators will evaluate the psychological function of adolescents and young adults, aged 12 to 20 years, with CF treated with ORKAMBI, and compare it to other adolescents and young CF adults not treated with ORKAMBI.
This is a cohort prospective study, with a 1 year follow-up.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's enrollment of 19 is below the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
CF Center patients
Exclusion Criteria:
Patients with proven CF (sweat-test \> 60 mEq, 2 DF508 CF causing mutations, 12 to 20 years, and treated with ORKAMBI)
Behavioral: Questionnaires
Patients not carrying 2 DF508 CF causing mutations, not treated with ORKAMBI, and not carrying the G551D, G178R, S549N, S549R, G551S, G1244E,S1251N, S1255P or G1349D mutation or treated with Kalydeco
Behavioral: Questionnaires
Answering 4 questionnaires : anxiety, depression, quality of life and adherence
Score of Generalized Anxiety Disorder-7 questionnaire (GAD-7)
to assess the impact on Anxiety
Time frame: 24 months
Score of Patient Health Questionnaire-9 (PHQ-9 )
To assess the impact on Depression
Time frame: 24 months
Scores of Cystic Fibrosis Questionnaire 14+ (CFQ 14+)
Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms (Quality of Life)
Time frame: 24 months
GIRERD Scale
The 6 items are assessed using a YES/NO answer .The scale was validated in patients with chronic disease to assess medication adherence
Time frame: 24 months
Plan to share: Undecided
This study is completed, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.
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Assistance Publique - Hôpitaux de Paris