retinol palmitate for Bronchopulmonary dysplasia

Inclusion criteria

• {"criterion_text":"- Very preterm infants with a GA <30 weeks (N=12): •\t7 extremely preterm infants ≤27 weeks + 6 days GA and •\t5 very preterm infants ≥28 weeks + 0 day GA and ≤29 weeks + 6 days GA to cover data from both sub-populations of preterm infants <30 weeks"}
• {"criterion_text":"- Enrollment between 24 hours (h) and 72 h of birth. Note: All genders and ethnicities are eligible for the study."}
• {"criterion_text":"- Informed consent must be obtained from the parents of the infants prior to enrollment in the study."}

Exclusion criteria

• {"criterion_text":"- Major congenital anomalies"}
• {"criterion_text":"- Intraventricular Hemorrhage (IVH) grade 2-4"}
• {"criterion_text":"- Infants at a high mortality risk, as judged by the hospital principal investigator (PI), for example •\tTerminal illness as evidenced by severe acidosis (pH < 7.0 for > 2 h) or persistent bradycardia (heart rate < 100 beats per minute) associated with hypoxia for > 2 h •\tCongenital nonbacterial infection with overt signs at birth"}
• {"criterion_text":"- Infants who are to receive vitamin A in a parenteral fat emulsion in doses exceeding recommendations for multivitamin preparations."}
• {"criterion_text":"- Infants born preterm due to maternal exposure to modifiable risk factors such as smoking, alcohol consumption, substance abuse, or other known teratogenic exposures."}

Primary endpoints

• {"endpoint_text":"- Primary Efficacy: Relative increase in serum retinol levels at Day 28 compared to baseline","definition_or_measurement_approach":"Serum retinol concentration measured at Days 0 and 28; primary objective states assessment of Retinol serum concentration at Days 0 and 28 in preterm infants."}
• {"endpoint_text":"- Safety: Incidence of Treatment Emergent Adverse Events (TEAEs) and Adverse Event of Specific Interest (AESI) throughout the study.","definition_or_measurement_approach":"Safety assessed by recording incidence of TEAEs and AESIs throughout study duration (Day 0 to week 36 PMA)."}
• {"endpoint_text":"- Safety: Occurrence of clinically significant changes in laboratory data, physical examination results, and vital signs values throughout the study.","definition_or_measurement_approach":"Clinical safety monitoring including laboratory data, physical exam findings and vital signs across study visits from Day 0 through follow-up (to 36 weeks PMA)."}

Secondary endpoints

• {"endpoint_text":"- Secondary efficacy:\tPercentage of subjects with relative increase of 60% in serum retinol levels between Day 0 and Day 28.","definition_or_measurement_approach":"Proportion of participants achieving ≥60% relative increase in serum retinol between Day 0 and Day 28 based on serum measurements."}
• {"endpoint_text":"- Secondary efficacy: Relative increase in serum retinol levels at Day 7 and Day 14 compared to baseline.","definition_or_measurement_approach":"Serum retinol concentration measured at Day 7 and Day 14 compared to baseline (Day 0)."}
• {"endpoint_text":"- Secondary efficacy: Percentage of subjects with need for respiratory support and/or oxygen at 28 days postnatal age.","definition_or_measurement_approach":"Proportion of participants requiring respiratory support and/or supplemental oxygen at 28 days postnatal age assessed by clinical records."}
• {"endpoint_text":"- Secondary efficacy:\tPercentage of subjects with need for respiratory support and/or oxygen at 36 weeks + 0 day PMA.","definition_or_measurement_approach":"Proportion of participants requiring respiratory support and/or oxygen at 36 weeks post-menstrual age (PMA) assessed at follow-up visit."}

Methods

• Distribution of recruitment flyers and posters (K2_Recruitment_Flyer and K1_Recruitment arrangements documents) targeted at parents of preterm infants in participating hospitals (Germany, Poland).
• Parent educational materials (K2_Recruitment_parent educational material, K2_Recruitment_DEU_parent educational material) provided to parents and caregivers.
• Email communications and brochures to site contacts/clinical staff (L2_Scout_Email Comm and L2_Scout_Brochure documents) to inform and engage clinical sites and parents.

Poland

Earliest CTIS Part Ii Submission Date

18-03-2025

Latest Decision Or Authorization Date

27-11-2025

Processing Time Days

254

Number Of Sites

1

Number Of Participants

8

Germany

Earliest CTIS Part Ii Submission Date

27-03-2025

Latest Decision Or Authorization Date

25-03-2026

Processing Time Days

363

Number Of Sites

1

Number Of Participants

12

Primary sponsor

Full Name

Aspire Pharma Limited

Organisation Type

Pharmaceutical company

Country Of Registered Address

United Kingdom

Contract research organisations

Name

Ergomed Group Limited

Responsibilities

Sponsor duties listed (codes: 1,11,12,2,5,6,7,9)

Third parties

• {"country":"United Kingdom","full_name":"Krystelis Limited","duties_or_roles":"Study documents redaction","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Germany","full_name":"Analytisches Zentrum Biopharm GmbH Berlin","duties_or_roles":"Sponsor duties listed (code: 4)","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Apcer Life Sciences Limited","duties_or_roles":"Sponsor duties listed (code: 8)","organisation_type":"Pharmaceutical company"}
• {"country":"Austria","full_name":"Lukas Heil-Betriebsstaette GmbH","duties_or_roles":"Final IMP release and distribution","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Ergomed Group Limited","duties_or_roles":"Sponsor duties listed (codes: 1,11,12,2,5,6,7,9)","organisation_type":"Pharmaceutical company"}