2023-508132-65-00
Not yet recruiting
Phase 2
Efficacy of methylprednisolone pulses in neuroendocrine cells hyperplasia of infancy: an early phase study (CORTICONEHI).
Assistance Publique Hopitaux De Paris23 sites in 1 country18 target enrollmentStarted: June 14, 2024Last updated:
Overview
- Phase
- Phase 2
- Status
- Not yet recruiting
- Enrollment
- 18
- Locations
- 23
- Primary Endpoint
- Number of children still requiring oxygen therapy at M18 (i.e. 18 months after the inclusion)
Overview
Brief Summary
To evaluate the efficacy on oxygen therapy need of a 6 months methylprednisolone pulses in NEHI patients at M18
Eligibility Criteria
- Ages
- 0 years to 17 years (0-17 Years)
- Accepts Healthy Volunteers
- Yes
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)
- •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)
Outcomes
Primary Outcomes
Number of children still requiring oxygen therapy at M18 (i.e. 18 months after the inclusion)
Number of children still requiring oxygen therapy at M18 (i.e. 18 months after the inclusion)
Secondary Outcomes
- Efficacy endpoint, number of children requiring only sleep oxygen at M18.
- Efficacy endpoint, number of children with a normal respiratory rate at rest at M18
- 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
- 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
- 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
- Safety endpoint, number of unexpected hospitalizations for a respiratory exacerbation between M0 and M18
- Safety endpoint, number of antibiotic courses for an infectious lung exacerbation between M0 and M18
- 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
- Safety endpoints, the number of patients with at least one an elevated fasting glycaemia before the first day of methylprednisolone pulses
- Exploratory endpoint, correlation between family impact PedsQL questionnaire and the newly described chILD-PQoL questionnaire at each time of measurement
- 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
Investigators
Pr Nadia NATHAN
Scientific
Assistance Publique Hopitaux De Paris
Study Sites (23)
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