Ignite Creation Date:
2024-07-17 @ 11:55 AM
Last Modification Date:
2024-10-26 @ 3:32 PM
Study NCT ID:
NCT06471556
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-24
First Post:
2024-06-13
Brief Title:
Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy An Early Phase Study
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
Official Title:
Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy An Early Phase Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
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:
CORTICONEHI
Brief Summary:
Childhood interstitial lung diseases chILD are a heterogeneous group of rare and severe disorders with an estimated prevalence of 1100000 Among them neuroendocrine cells hyperplasia of infancy NEHI also called persistent tachypnoea of infancy PTI is one of the most common aetiology up to 16 of the cases NEHI involves young infants median age at onset 3 to 6 months with tachypnoea hypoxemia crackles retractions failure to thrive and specific localizations of ground glass opacities GGO on chest CT-scan paramediastinal areas and anterior lobes right middle lobe and lingula At diagnosis most patients 50 to 100 require oxygen supplementation that usually lasts for months to years sometimes associated with nutritional support with eventual enteral nutrition NEHI is believed to be related to an increased number of neuroendocrine cells in airway epithelial area These cells are abundant in foetal life when they play a role in regulating the lung development and decrease before birth
There is no specific treatment for NEHI The main treatment of chILD is corticosteroids However in NEHI their efficacy is matter of debate There is only a few NEHI cases series or cohorts all over the world accounting for a maximum of 500 reported cases within only retrospective studies Among them United States and Argentina teams report supportive care only oxygen therapy and nutritional support whereas other teams like the French ones largely uses IV corticosteroid pulses
Unlike the majority of chILD NEHI prognosis is usually good However at school-age 26 of the patients remain symptomatic or have an abnormal lung function Moreover oxygen therapy significantly affects quality of life QoL of the children with ILD -1043100 points p002 but also QoL and mood of their parents unpublished data
The present study hypothesis that corticosteroids are associated with a reduction of the length of oxygen support in infants with NEHI
There is no specific treatment for NEHI The main treatment of chILD is corticosteroids However in NEHI their efficacy is matter of debate There is only a few NEHI cases series or cohorts all over the world accounting for a maximum of 500 reported cases within only retrospective studies Among them United States and Argentina teams report supportive care only oxygen therapy and nutritional support whereas other teams like the French ones largely uses IV corticosteroid pulses
Unlike the majority of chILD NEHI prognosis is usually good However at school-age 26 of the patients remain symptomatic or have an abnormal lung function Moreover oxygen therapy significantly affects quality of life QoL of the children with ILD -1043100 points p002 but also QoL and mood of their parents unpublished data
The present study hypothesis that corticosteroids are associated with a reduction of the length of oxygen support in infants with NEHI
Detailed Description:
This study aims at assessing the beneficial effect of IV methylprednisolone pulses on oxygen therapy in infants with NEHI It applies to all hypoxic NEHI patients aged below 12 months and followed in one of the centres of the RespiRare network 18 patients will be recruited
CORTICO-NEHI is designed as an early phase study following an AHerns Single Stage Phase II design
All the patients are included in a single group receiving as a standard of care a maximum of 6 IV pulses of 3 days each
12 visits will be performed as a standard of care
Screening visit M-1 at maximum follow-up routine visit usually the hospitalization for NEHI diagnosis If the patient is eligible parents are informed of the study and an informed note is given Information on the study protocol is given to obtain their written consent as soon as possible and at the latest at the inclusion
Inclusion M0 After checking of the inclusion criteria and the absence of exclusion criteria information and collection of parental consent Following these investigations the patients first IV methylprednisolone pulse D1 is administrated within the limit of 10 days
Treatment visits M0 - M5 During a hospitalization 6 pulses are performed at a 4 weeks interval - 10 days Each pulse is a 3-days-6h-perfusions of Methylprednisolone 500mg and 120mg 10mgkgday diluted in 50ml of saline under supervision of SpO2 heart rate blood pressure 30min If oxygen can be stopped 2 oxymetries with a 1-week interval - 10 days The endpoint is reached and the methylprednisolone pulses are definitively stopped
Follow-up visits M6 M9 M12 M15 and M18
For the research needs
CT scan and Echocardiography will be done at inclusion if not performed in the past 3 months
QoL questionnaires will be performed at inclusion M6 M12 and M18
Dipstick for sugar will be performed at M6 M9 M12 M15 M18 in addition to M0 - M5 according to the standard of care
CORTICO-NEHI is designed as an early phase study following an AHerns Single Stage Phase II design
All the patients are included in a single group receiving as a standard of care a maximum of 6 IV pulses of 3 days each
12 visits will be performed as a standard of care
Screening visit M-1 at maximum follow-up routine visit usually the hospitalization for NEHI diagnosis If the patient is eligible parents are informed of the study and an informed note is given Information on the study protocol is given to obtain their written consent as soon as possible and at the latest at the inclusion
Inclusion M0 After checking of the inclusion criteria and the absence of exclusion criteria information and collection of parental consent Following these investigations the patients first IV methylprednisolone pulse D1 is administrated within the limit of 10 days
Treatment visits M0 - M5 During a hospitalization 6 pulses are performed at a 4 weeks interval - 10 days Each pulse is a 3-days-6h-perfusions of Methylprednisolone 500mg and 120mg 10mgkgday diluted in 50ml of saline under supervision of SpO2 heart rate blood pressure 30min If oxygen can be stopped 2 oxymetries with a 1-week interval - 10 days The endpoint is reached and the methylprednisolone pulses are definitively stopped
Follow-up visits M6 M9 M12 M15 and M18
For the research needs
CT scan and Echocardiography will be done at inclusion if not performed in the past 3 months
QoL questionnaires will be performed at inclusion M6 M12 and M18
Dipstick for sugar will be performed at M6 M9 M12 M15 M18 in addition to M0 - M5 according to the standard of care
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 |
|---|---|---|---|
| 2023-508132-65-00 | CTIS | None | None |