Phase 1 Safety Trial of Recombinant Surfactant Protein D to Prevent Neonatal Chronic Lung Disease

18/0564

The purpose of this study is to identify the safest dose of recombinant surfactant protein D (drug name: rfhSP-D) that can be administered to preterm infants born at less than 28 weeks gestation, and to help identify whether this can prevent the development of neonatal chronic lung disease.

2023-12-01

2024-07-01

2024-12-01

Not yet recruiting

Early phase I

24

Participant Inclusion Criteria: 1. Inborn infants born at between 23 weeks and 0 days and \<28 weeks and 0 days gestation. 2. Infant must be intubated or planned to be intubated for respiratory distress at time of eligibility check, and this should be done within 12 hours from time of birth. 3. Receiving standard surfactant therapy 4. Clinically stable on mechanical ventilation. Stability is defined at the time of IMP instillation and is defined below. 5. Written informed consent from parents/guardians/person with legal responsibility Definition of stability: 1. Blood gases within the normal range for preterm infants (pH\>7.20; paCO2 \<60mmHg) 2. Mean blood pressure with or without inotropic support at at least gestational age or above (mmHg) 3. No evidence of a pneumothorax 4. Clinical observations within acceptable range for an infant of that gestational age 5. No stability concerns from the attending neonatologist Participant Exclusion Criteria: 1. Congenital anomalies i.e any major antenatal diagnosed congenital abnormalities such as congenital heart disease, suspected or known chromosomal abnormalities 2. Parents/legal guardians unable to give consent due to learning or other difficulties 3. Infants requiring only CPAP support without the need for surfactant replacement therapy, i.e. without endotracheal intubation 4. Infants born in very poor condition and judged too sick or unstable to be included (high risk of mortality) in an experimental first in human study, for example infants that are requiring maximal intensive care therapy and have findings such as a grade IV intraventricular haemorrhage that is likely to be life limiting. 5. Infants that are born out of the participating site. 6. Participation in any other interventional study (participation in an observational study is permissible).

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'A Bayesian Continual Reassessment Method (CRM) will be used for the RESPONSE trial to inform how the IMP dose should be adapted for the next cohort based on past trial data. The CRM is a model-based design that uses a statistical model to estimate the risk of dose limiting events (DLE) per dose level. The target level of DLEs is set at 20% The CRM model does not allow dose-skipping. The recommended phase 2 dose in terms of safety (efficacy will also be taken into account) will be the highest dose level that has an estimated probability of DLE closest but below the target DLE level of 20%.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 24, 'type': 'ESTIMATED'}}

2023-11-30