Ongoing Clinical Trials for Neuroendocrine Cell Hyperplasia of Infancy
Currently, there is 1 ongoing clinical trial investigating treatment options for neuroendocrine cell hyperplasia of infancy, a rare lung condition affecting infants. This trial is taking place in France and is testing the effectiveness of methylprednisolone, a steroid medication, in reducing the need for oxygen therapy in affected children.
Clinical trial locations
Study on the Effects of Methylprednisolone in Infants with Neuroendocrine Cell Hyperplasia
This clinical trial focuses on a rare lung condition that affects infants, causing an overgrowth of certain cells in the lungs and leading to breathing difficulties. The condition requires many affected children to use oxygen therapy to help them breathe properly.
Who can participate:
- Infants younger than 12 months old
- Children diagnosed with the condition either through a clinical scoring system combined with chest imaging showing characteristic patterns, or confirmed by a lung tissue sample showing increased neuroendocrine cells
- Infants who currently need oxygen therapy while awake or asleep
- Those receiving care at participating centers in the RespiRare network in France
- Parents or legal guardians must provide written consent for their child to participate
Who cannot participate:
- Infants with other serious health conditions that could interfere with the study
- Those currently participating in another clinical trial
- Children with recent infections or illnesses that might affect results
- Infants who cannot follow study procedures or attend required visits
- Those with allergies to the study medication or similar drugs
- Children taking medications that might interfere with the treatment
- Those with a history of not following medical treatments
- Any condition that doctors believe would make participation unsafe
Main goal and approach:
The trial aims to evaluate how effective methylprednisolone is in reducing the need for oxygen therapy over 18 months. Methylprednisolone is a type of steroid medication that helps reduce inflammation in the body. It works by blocking the release of substances that cause inflammation, thereby helping to calm the immune response.
The medication will be given through an intravenous infusion (directly into a vein) in pulses over a 6-month treatment period. Some participants may receive a placebo, which contains no active medication, to allow researchers to compare the effects of the actual treatment.
Researchers will track several important outcomes, including how many children still need oxygen therapy after 18 months, whether some children only need oxygen during sleep, and changes in breathing patterns. The study will also monitor safety by checking for unexpected hospital visits, antibiotic use for lung infections, blood pressure, and blood sugar levels. Additionally, the impact on quality of life for both the children and their families will be assessed using specific questionnaires.
The trial includes lung imaging at the end of the study period to evaluate any changes in the lungs. The study is expected to conclude by January 2028.
Summary
Currently, only one clinical trial is actively investigating treatment options for neuroendocrine cell hyperplasia of infancy. This trial is being conducted in France and focuses specifically on methylprednisolone, a steroid medication with anti-inflammatory properties. The trial represents an important step in understanding potential treatment options for this rare condition, as it aims to determine whether this medication can reduce the dependency on oxygen therapy in affected infants. The study’s comprehensive approach includes not only measuring clinical outcomes but also evaluating the impact on families’ quality of life and carefully monitoring safety throughout the treatment period.
