Ignite Creation Date:
2024-05-06 @ 8:25 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06370962
Status:
RECRUITING
Last Update Posted:
2024-06-03
First Post:
2024-02-19
Brief Title:
Circadian Rhythm Disorders in Children With Cystic Fibrosis Under CFTR Cystic Fibrosis Transmembrane Conductance Regulator Modulators
Sponsor:
Hospices Civils de Lyon
Organization:
Hospices Civils de Lyon
Study Overview
Official Title:
Overview of Circadian Rhythm Disorders in Children With Cystic Fibrosis in the Era of CFTR Cystic Fibrosis Transmembrane Conductance Regulator Modulators
Status:
RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
CHRONO-MUCO
Brief Summary:
Cystic fibrosis CF is a rare disease affecting one out of 4500 newborns in France INSERM 2021 Despite major advances in patient care over the past two decades with significant improvements in life expectancy cystic fibrosis remains a pathology that considerably impairs quality of life
Several studies have reported the possibility of respiratory and non-respiratory sleep disorders SD in patients with CF Respiratory disorders are reported to affect 30 of children with CF Barbosa 2020 Among non-respiratory SD sleep onset and maintenance insomnia are well known in these patients while chronotype abnormalities circadian rhythm disorders are understudied Chronotype refers to a persons tendency to be more efficient in the morning or evening
The existence of chronotype abnormalities has been suggested in CF patients but no precise data are available Louis 2022 The involvement of CFTR Cystic Fibrosis Transmembrane Conductance Regulator protein dysfunction in the central nervous system CNS has been hypothesized as a contributory factor In vivo in a mouse model of CF dysregulation of clock genes such as Clock Cry2 and Per2 was found in the CNS Barbato 2019 Among them certain genes such as Rev-erbĪ± could regulate endobronchial inflammation and contribute to the severity of respiratory pathology All in all chronotype abnormalities could be at the origin of sleep debt impaired cognitive functions or metabolic disturbances
In the era of highly effective modulator therapy HEMT for the treatment of CF the impact of these new therapies on chronotype has been understudied Assuming that chronotype abnormalities are a direct consequence of CFTR protein dysfunction in the retina and anterior hypothalamus HEMT should improve sleep quality However between 20 and 30 of adult and pediatric patients express an increase in chronotype abnormalities following initiation of treatment
Paradoxically the perceived gain in respiratory quality of life is counterbalanced by the occurrence of these disorders Some patients would effectively reverse their treatment in order to limit the phenomenon A single polysomnographic study evaluated the effect of HEMT Kaftrio-Kalydeco on sleep in adults with CF Welsner 2022 After 3 months of treatment patients had a significant reduction in respiratory events with no change in total sleep time sleep efficiency or sleep architecture Chronotype was not mentioned Currently no studies on chronotype in children or adults with CF have been carried out Our hypothesis is that CF patients treated with HEMT would develop an abnormal chronotype of late sleep onset
The aim of this study is to evaluate the chronotype of children with CF treated with HEMT Chronotype abnormalities could have major consequences for quality of life the immune system cognitive functions and metabolism Systematic detection of these disorders via anamnesis followed by diagnosis by questionnaire actimetrics andor urinary melatonin dosage would enable their early management starting with the reversal of Kaftrio-Kalydeco intake between morning and evening
Several studies have reported the possibility of respiratory and non-respiratory sleep disorders SD in patients with CF Respiratory disorders are reported to affect 30 of children with CF Barbosa 2020 Among non-respiratory SD sleep onset and maintenance insomnia are well known in these patients while chronotype abnormalities circadian rhythm disorders are understudied Chronotype refers to a persons tendency to be more efficient in the morning or evening
The existence of chronotype abnormalities has been suggested in CF patients but no precise data are available Louis 2022 The involvement of CFTR Cystic Fibrosis Transmembrane Conductance Regulator protein dysfunction in the central nervous system CNS has been hypothesized as a contributory factor In vivo in a mouse model of CF dysregulation of clock genes such as Clock Cry2 and Per2 was found in the CNS Barbato 2019 Among them certain genes such as Rev-erbĪ± could regulate endobronchial inflammation and contribute to the severity of respiratory pathology All in all chronotype abnormalities could be at the origin of sleep debt impaired cognitive functions or metabolic disturbances
In the era of highly effective modulator therapy HEMT for the treatment of CF the impact of these new therapies on chronotype has been understudied Assuming that chronotype abnormalities are a direct consequence of CFTR protein dysfunction in the retina and anterior hypothalamus HEMT should improve sleep quality However between 20 and 30 of adult and pediatric patients express an increase in chronotype abnormalities following initiation of treatment
Paradoxically the perceived gain in respiratory quality of life is counterbalanced by the occurrence of these disorders Some patients would effectively reverse their treatment in order to limit the phenomenon A single polysomnographic study evaluated the effect of HEMT Kaftrio-Kalydeco on sleep in adults with CF Welsner 2022 After 3 months of treatment patients had a significant reduction in respiratory events with no change in total sleep time sleep efficiency or sleep architecture Chronotype was not mentioned Currently no studies on chronotype in children or adults with CF have been carried out Our hypothesis is that CF patients treated with HEMT would develop an abnormal chronotype of late sleep onset
The aim of this study is to evaluate the chronotype of children with CF treated with HEMT Chronotype abnormalities could have major consequences for quality of life the immune system cognitive functions and metabolism Systematic detection of these disorders via anamnesis followed by diagnosis by questionnaire actimetrics andor urinary melatonin dosage would enable their early management starting with the reversal of Kaftrio-Kalydeco intake between morning and evening
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2024-A00343-44 | OTHER | ID-RCB | None |