A Clinical Efficacy and Safety Study of OHB-607 in Preventing Chronic Lung Disease in Extremely Premature Infants

Study Overview
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Brief Summary
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The purpose of this study is to determine if an investigational drug can reduce the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants receiving standard neonatal care alone.

Official Title
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A Phase 2b, Multicenter, Randomized, Open-label, Two-Arm Study to Evaluate the Clinical Efficacy and Safety of OHB-607 in Preventing Chronic Lung Disease in Extremely Premature Infants Compared to Standard Neonatal Care

Conditions
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Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage, Retinopathy of Prematurity (ROP)

Intervention / Treatment
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* Drug: OHB-607


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Written informed consents and/or assents must be signed and dated by the participant's parent(s) prior to any study related procedures. The informed consent and any assents for underage parents must be approved by the IRB/IEC (in accordance with local regulations). Written informed consents and/or assents must be signed and dated by the participant's birth mother prior to providing study-related information related to birth mother medical history, pregnancy and the birth of the participant. The informed consent and any assents for underage birth mothers must be approved by the IRB/IEC (in accordance with local regulations). Subjects must be between 23 weeks +0 days and 27 weeks +6 days GA, inclusive. Exclusion Criteria: Detectable major (or severe) congenital malformation identified before randomization. Known or suspected chromosomal abnormality, genetic disorder, or syndrome, identified before randomization, according to the investigator's opinion. Hypoglycemia at Baseline (blood glucose less than (<) 45 milligrams per deciliter [mg/dL] or 2.5 milli moles per liter [mmol/L]) which persists in spite of glucose supplementation, to exclude severe congenital abnormalities of glucose metabolism. Clinically significant neurological disease identified before randomization according to cranial ultrasound (hemorrhages confined to the germinal matrix are allowed) and investigator's opinion. Any other condition or therapy that, in the investigator's opinion, may pose a risk to the participant or interfere with the participant's potential compliance with this protocol or interfere with interpretation of results. Current or planned participation in a clinical study of another investigational study treatment, device, or procedure (participation in non-interventional studies is permitted on a case-by-case basis). The participant or participant's parent(s) is/are unable to comply with the protocol or is unlikely to be available for long-term follow-up as determined by the investigator. Birth mother with active COVID-19 infection at birth or a history of severe COVID-19 infection (requiring intensive care hospitalization) during pregnancy.

Ages Eligible for Study
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Minimum Age: 0 Hours
Maximum Age: 24 Hours

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OHB-607<br>Participants will receive continuous IV infusion of OHB-607 through from birth up to PMA 29 weeks +6 days. | Drug: OHB-607<br>* Participants will receive intravenous infusion of OHB-607 from birth up to PMA 29 weeks + 6 days.<br>* Other names: Mecasermin Rinfabate;|
| No Intervention: Standard Neonatal Care<br>Standard neonatal care alone will be provided. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Reduction in the incidence of severe BPD at 36 weeks (±3 days) PMA, or death, whichever comes first as compared to the SNC group | Severe BPD is defined by the modified NICHD severity grading | Baseline through 36 weeks postmenstrual age (PMA) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence of severe (Grade 3 and 4) IVH at 36 weeks PMA, as assessed by cranial ultrasound as compared to the SNC group | Severe IVH as classified according to the Volpe criteria | Baseline through 36 weeks postmenstrual age (PMA) | 
| Incidence and severity of BPD | BPD severity is defined by the modified NICHD severity grading | Baseline through 36 weeks postmenstrual age (PMA) | 
| Incidence and severity of IVH | IVH grade as classified according to the Volpe criteria | Baseline through 36 weeks postmenstrual age (PMA) | 
| Neurodevelopment outcomes | Neurodevelopmental impairment, Physical and cognitive development will be measured by standardised questionnaires | From 6 months CA through 24 months CA | 
| Incidence of Retinopathy of Prematurity (ROP) | ROP is classified according to the International Classification | Baseline through 40 weeks PMA | 
| Mortality from birth through to 24 months CA | Mortality rates from >12 hours after birth through 24 months CA | From birth through 24 months CA | 
| Exposure-response Pharmacokinetics/Pharmacodynamics relationships | Relationship between IGF-1 exposure and study endpoints | Baseline through 40 weeks PMA |