RecruitingRare disease

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

Verified siteRegistered drugNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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.
Step 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.
Step 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.
Step 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.
Step 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 trial?

6 criteria

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 trial?

10 criteria

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.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Assistance Publique Hopitaux De Marseille
Verified
Marseille, France
Besancon University Hospital Center
Verified
Besançon, France
CHRU De Nancy
Responsive
Vandoeuvre Les Nancy, France

Trial locations

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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.

What is already known about the treatment

Methylprednisolone – This medication is administered intravenously and is currently being studied for its effectiveness in treating neuroendocrine cell hyperplasia of infancy (NEHI). It is a corticosteroid, widely recognized in medical literature for its anti-inflammatory and immunosuppressive properties. The main therapeutic indication in this trial is to assess its impact on reducing the need for oxygen therapy in NEHI patients. At the molecular level, methylprednisolone works by inhibiting the release of substances in the body that cause inflammation, thereby modulating the immune response.

Investigated diseases

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.