Study on the Effects of Methylprednisolone in Infants with Neuroendocrine Cell Hyperplasia

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What is this study about?

This clinical trial is focused on studying a rare lung condition called Neuroendocrine Cell Hyperplasia of Infancy (NEHI). NEHI is a disease that affects infants and involves an overgrowth of certain cells in the lungs, which can lead to breathing difficulties. The study will use a medication called Methylprednisolone, which is a type of steroid that can help reduce inflammation in the body. The medication will be given as a powder that is mixed into a solution and then injected into the bloodstream through a vein.

The purpose of the study is to evaluate how effective Methylprednisolone is in reducing the need for oxygen therapy in infants with NEHI over a period of 18 months. Participants in the study will receive treatment with Methylprednisolone for six months. The study will also involve monitoring the infants’ breathing and overall health to see if there are improvements in their condition. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of the treatment.

Throughout the study, researchers will track various health outcomes, such as the number of children who still need oxygen therapy after 18 months, and any changes in their breathing patterns. The study will also look at the impact of the treatment on the quality of life for both the children and their families. Safety will be closely monitored, including any unexpected hospital visits or changes in blood pressure. The study aims to provide valuable information on the potential benefits of Methylprednisolone for infants with NEHI.

1 joining the study

The study involves infants under 12 months diagnosed with neuroendocrine cells hyperplasia of infancy (NEHI).

Diagnosis is based on specific clinical criteria and imaging results.

Participation requires written consent from the legal representative.

2 treatment initiation

The treatment involves the administration of methylprednisolone through intravenous infusion.

The treatment is given in pulses over a period of 6 months.

The objective is to assess the need for oxygen therapy after 18 months.

3 oxygen therapy assessment

The primary goal is to determine the number of children still requiring oxygen therapy 18 months after starting the study.

Secondary assessments include the need for oxygen only during sleep and the respiratory rate at rest.

4 safety and efficacy evaluation

Safety is monitored by tracking unexpected hospitalizations and antibiotic use for lung infections.

Blood pressure and blood sugar levels are checked during the treatment period.

The impact on quality of life for patients and families is evaluated using specific questionnaires.

5 follow-up and conclusion

The study concludes with a final assessment of lung imaging results and overall health status at 18 months.

The study is expected to end by January 2028.

Who Can Join the Study?

The infant must be younger than 12 months old.
The infant must have a diagnosis of NEHI (Neuroendocrine Cells Hyperplasia of Infancy). This can be confirmed by:
- A clinical score called the Liptzin score of 7 or more out of 10, along with a specific pattern seen on a chest CT scan (a detailed imaging test) showing areas called ground glass opacities in certain parts of the lungs.
- Or, a suspicion based on clinical and CT findings, confirmed by a lung biopsy (a small sample of lung tissue) showing an increased number of neuroendocrine cells (a type of cell in the airways).
The infant must need oxygen therapy (extra oxygen) when awake or asleep, as recommended for children.
The infant must be receiving care at one of the participating centers in the RespiRare network.
The legal representative(s) of the infant must provide written informed consent for participation in the study.

Who Cannot Join the Study?

Patients who have any other serious health conditions that could interfere with the study.
Patients who are currently participating in another clinical trial.
Patients who have had a recent infection or illness that could affect the study results.
Patients who are unable to follow the study procedures or attend the required visits.
Patients who have allergies or reactions to the study medication or similar drugs.
Patients who are taking medications that might interfere with the study treatment.
Patients who have a history of non-compliance with medical treatments.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse.
Patients who have any condition that the study doctors believe would make it unsafe for them to participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
University Hospital Of Clermont-Ferrand	Clermont Ferrand	France
Centre Hospitalier Intercommunal Creteil	Creteil	France
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France
Centre Hospitalier Universitaire De Lille	Lille	France
Centre Hospitalier Regional Et Universitaire De Brest	Brest	France
Oncopole Claudius Regaud	Toulouse	France
CHU Grenoble Alpes	La Tronche	France

Other Sites

Site Name	City	Country
Centre Hospitalier Universitaire Rouen	Rouen	France
Centre Hospitalier Universitaire De La Reunion	St Denis	France
Fondation Lenval Nice	Nice	France
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Rennes	Rennes	France
Centre Hospitalier Lyon Sud	Pierre Benite	France
Hopital Beaujon	Clichy	France
Centre Hospitalier Universitaire De Caen Normandie	Caen	France
Cqrrzf Hbairpsxfjb Uolrcksrxnxsw Rakct	Reims	France
Cjkvba Huicfwngvyw Ej Udbhswisseigi Dl Lbvcyik	Limoges	France
Awpxhsojrc Pqyagpar Hummiznb Df Mazbnolqu	Marseille	France
Bmfitvwe Ucoxctsiwp Heyvpxnv Cacpyr	Besançon	France
Chfdpd Hmvbamrcroa Raqfwbzy Uqcmbrcvaivby Dw Tjtun	Tours	France
Cbah Dn Nmskj	Vandoeuvre Les Nancy	France
Cqrfls Hqmmjffovku Rtvkuovx Dzcsnabbjeaeha	Angers	France
Hlcupyuc Ujrdcedwamyjbr Styweviuok &cqfdzn Hqtfude da Hovxykvhkkh	STRASBOURG, Alsace	France

Trial status

Country	Status	Recruitment Start
France	Recruiting	15.04.2024

Trial locations

Investigated drugs:

Methylprednisolone is a medication used in this clinical trial to assess its effectiveness in treating neuroendocrine cell hyperplasia of infancy (NEHI). It is a type of corticosteroid that helps reduce inflammation in the body. In this study, the focus is on determining whether methylprednisolone can decrease the need for oxygen therapy in patients with NEHI over a period of 18 months.

Investigated diseases:

Neuroendocrine cell hyperplasia of infancy

Neuroendocrine Cell Hyperplasia of Infancy – This is a rare lung disease that affects infants, characterized by an abnormal increase in neuroendocrine cells in the lungs. It typically presents with symptoms such as rapid breathing and low oxygen levels, often requiring supplemental oxygen. The condition is usually identified in the first few months of life and can lead to persistent respiratory issues. Over time, some children may experience improvement in symptoms, while others may continue to need oxygen support, especially during sleep. The progression of the disease can vary, with some children showing a decrease in lung abnormalities as they grow older. Monitoring and supportive care are essential to manage the respiratory symptoms associated with this condition.

Trial ID:

2023-508132-65-00

Protocol code:

APHP230821

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on the Effects of Methylprednisolone in Infants with Neuroendocrine Cell Hyperplasia

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