METHYLPREDNISOLONE HYDROGEN SUCCINATE for Neuroendocrine cell hyperplasia of infancy

Inclusion criteria

• {"criterion_text":"- Infant aged under 12 months\n- 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)\n- Oxygen requirement (awake and/or asleep) based on the usual pediatric recommendations\n- Followed in one of the RespiRare participating centers\n- Written informed consent of the holder(s) of its legal representative at the inclusion"}

Exclusion criteria

• {"criterion_text":"- Other cause of chILD assessed by lab biology tests, genetic analysis for surfactant genes (if available), bronchoalveolar lavage, and/or lung biopsy\n- Patient treated with IV methylprednisolone pulses before (any time)\n- Diabetes\n- Uncontrolled arterial hypertension\n- Absence of Health care insurance\n- Ongoing infection\n- Immunization with a live attenuated vaccine within the past two weeks\n- Long term treatment with Azithromycin and/or Hydroxychloroquine\n- Patients already included in an interventional study (RIPH1, clinical investigation or clinical trial)"}

Primary endpoints

• {"endpoint_text":"- Number of children still requiring oxygen therapy at M18 (i.e. 18 months after the inclusion)","definition_or_measurement_approach":"Oxygen need will be assessed by oximetry during sleep and awake (≥2 hours awake and ≥6 hours sleep)."}

Secondary endpoints

• {"endpoint_text":"- Efficacy endpoint, number of children requiring only sleep oxygen at M18.","definition_or_measurement_approach":"Assessed at M18 (timepoint specified)."}
• {"endpoint_text":"- Efficacy endpoint, number of children with a normal respiratory rate at rest at M18","definition_or_measurement_approach":"Assessed at M18 (timepoint specified)."}
• {"endpoint_text":"- Efficacy endpoint, difference of Fan’s severity score assessing the severity of children with ILD (respiratory symptoms, SpO2<90% at sleep or exertion, SpO2<90% at rest and pulmonary hypertension) between M0) and M18","definition_or_measurement_approach":"Difference in Fan's severity score between baseline (M0) and M18; Fan score assesses respiratory symptoms, SpO2<90% at sleep/exertion, SpO2<90% at rest and pulmonary hypertension (see protocol chapter 8 for score details)."}
• {"endpoint_text":"- Efficacy endpoint, patients and family impact PedsQL and the chILD QoL scores; the chILDPQoL score, a parents QoL score (submitted for publication) at M0 and M18","definition_or_measurement_approach":"Quality of life measured by PedsQL and chILD QoL (chILDPQoL) at M0 and M18."}
• {"endpoint_text":"- Efficacy endpoint, number of patients requiring at least one enteral nutrition episode (based on the physician appreciation in case of abnormal weight curve) between M0 and M18","definition_or_measurement_approach":"Number of patients with ≥1 enteral nutrition episode between M0 and M18, based on physician assessment of weight curve."}
• {"endpoint_text":"- Safety endpoint, number of unexpected hospitalizations for a respiratory exacerbation between M0 and M18","definition_or_measurement_approach":"Count of unexpected hospitalizations for respiratory exacerbation between M0 and M18."}
• {"endpoint_text":"- Safety endpoint, number of antibiotic courses for an infectious lung exacerbation between M0 and M18","definition_or_measurement_approach":"Count of antibiotic courses for infectious lung exacerbations between M0 and M18."}
• {"endpoint_text":"- Safety endpoint, at M6, the number of patients with at least once systolic or diastolic arterial blood pressure (AP) over 2SD for the age at the days of methylprednisolone pulses","definition_or_measurement_approach":"At M6, number of patients with at least one systolic or diastolic blood pressure >2 SD for age on days of methylprednisolone pulses."}
• {"endpoint_text":"- Safety endpoints, the number of patients with at least one an elevated fasting glycaemia before the first day of methylprednisolone pulses","definition_or_measurement_approach":"Number of patients with at least one elevated fasting glycaemia before first day of methylprednisolone pulses."}
• {"endpoint_text":"- Exploratory endpoint, correlation between family impact PedsQL questionnaire and the newly described chILD-PQoL questionnaire at each time of measurement","definition_or_measurement_approach":"Correlation analysis between PedsQL family impact and chILD-PQoL at each measurement timepoint."}
• {"endpoint_text":"- Exploratory endpoint, number of patients with an extension, a stability, a decrease or an absence of the GGO lesions on the CT-scan at M6 and M18","definition_or_measurement_approach":"Radiological assessment of ground glass opacity (GGO) lesions on thoracic CT at M6 and M18 (extension, stability, decrease, or absence)."}

Other endpoints

• {"endpoint_text":"- Exploratory endpoint, correlation between family impact PedsQL questionnaire and the newly described chILD-PQoL questionnaire at each time of measurement","definition_or_measurement_approach":"Correlation analysis between PedsQL family impact and chILD-PQoL at each measurement timepoint."}
• {"endpoint_text":"- Exploratory endpoint, number of patients with an extension, a stability, a decrease or an absence of the GGO lesions on the CT-scan at M6 and M18","definition_or_measurement_approach":"Radiological assessment of GGO lesions on thoracic CT at M6 and M18."}

France

Earliest CTIS Part Ii Submission Date

13-05-2024

Latest Decision Or Authorization Date

08-01-2026

Processing Time Days

605

Number Of Sites

27

Number Of Participants

18

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France