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Tests treatment safety and results for Neuroendocrine Cell Hyperplasia of Infancy
Official title Efficacy of Methylprednisolone Pulses in Neuroendocrine Celles Hyperplasia of Infancy : An Early Phase Study
ClinicalTrials.gov ID: NCT06471556
What this study is testing
What is IV Methylprednisolone pulses?
IV Methylprednisolone pulses is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for neuroendocrine cell hyperplasia of infancy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Childhood interstitial lung diseases (chILD) are a heterogeneous group of rare and severe disorders with an estimated prevalence of 1/100,000. 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).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 12
You may be able to join if
- Infant aged under 12 months
- NEHI diagnosis based on:
- The recently validated clinical Liptzin score ≥7/10 associated with a suggestive thoracic CT pattern with ground glass opacities confined to middle...
- a clinical and thoracic CT suspicion and a lung biopsy showing an increased number of neuroendocrine cells in the epithelial airways area (at least...
- Oxygen requirement (awake and/or asleep) based on the usual pediatric recommendations (see section 4.1.1)
You likely can't join if
- Other cause of chILD assessed by lab biology tests, genetic analysis for surfactant genes (if available), bronchoalveolar lavage, and/or lung biopsy.
- Patient treated with IV methylprednisolone pulses before (any time)
- Diabetes
- Uncontrolled arterial hypertension
- Absence of Health care insurance
- Ongoing infection
See the full eligibility criteria
- Infant aged under 12 months
- NEHI diagnosis based on:
- The recently validated clinical Liptzin score ≥7/10 associated with a suggestive thoracic CT pattern with ground glass opacities confined to middle lobe, lingula, and paramediastinal lung areas OR
- a clinical and thoracic CT suspicion and a lung biopsy showing an increased number of neuroendocrine cells in the epithelial airways area (at least one bronchiole with at least 10% of neuroendocrine cells)
- Oxygen requirement (awake and/or asleep) based on the usual pediatric recommendations (see section 4.1.1)
- Followed in one of the RespiRare participating centers
- Written informed consent of the holder(s) of its legal representative at the inclusion
- Other cause of chILD assessed by lab biology tests, genetic analysis for surfactant genes (if available), bronchoalveolar lavage, and/or lung biopsy.
- Patient treated with IV methylprednisolone pulses before (any time)
- Diabetes
- Uncontrolled arterial hypertension
- Absence of Health care insurance
- Ongoing infection
- Immunization with a live attenuated vaccine within the past two weeks
- Long term treatment with Azithromycin and/or Hydroxychloroquine
- Patients already included in an treatment study (RIPH1, clinical investigation or clinical trial)
The study team makes the final eligibility decision.
Where it's taking place
- Paris, France
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 12 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Paris, France. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.