Efficacy of methylprednisolone pulses in neuroendocrine cells hyperplasia of infancy: an early phase study (CORTICONEHI).

Phase 1

• Conditions: EUROENDOCRINE CELLS HYPERPLASIA OF INFANCY; MedDRA version: 21.1Level: PTClassification code: 10072968Term: Neuroendocrine cell hyperplasia of infancy Class: 100000004855; MedDRA version: 20.0Level: SOCClassification code: 10038738Term: Respiratory thoracic and mediastinal disorders Class: 13; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: CTIS2023-508132-65-00
• Lead Sponsor: Assistance Publique Hopitaux De Paris

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-03-04
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 19

Inclusion 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, Followed in one of the RespiRare participating centers, Written informed consent of the holder(s) of its legal representative at the inclusion

Exclusion Criteria

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 interventional study (RIPH1, clinical investigation or clinical trial)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified