A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants

A Phase 2b, Multicenter, Randomized, Open-label, Two-Arm Study to Evaluate the Clinical Efficacy and Safety of OHB-607 Compared to Standard Neonatal Care for the Prevention of Bronchopulmonary Dysplasia, the Most Common Cause of Chronic Lung Disease of Prematurity

The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in extremely premature infants, as compared to extremely premature infants receiving standard neonatal care alone.

Study Overview

• Status: Recruiting
• Conditions: Intraventricular Hemorrhage; Bronchopulmonary Dysplasia; Chronic Lung Disease of Prematurity; Retinopathy of Prematurity (ROP)
• Intervention / Treatment: Drug: OHB-607

• Study Type: Interventional
• Enrollment (Estimated): 338
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: OHB Contact; Email: CMO@Oakhillbio.com

Study Locations

• Australia
  • Parkville, Australia, 3052
    • Recruiting
    • Royal Women's Hospital
  • South Brisbane, Australia, Qld 4101
    • Recruiting
    • Mater Misericordiae Limited
  • New South Wales
    • Randwick, New South Wales, Australia, 2031
      • Recruiting
      • Royal Hospital for Women
• Canada
  • Toronto, Canada, M5G 1X5
    • Withdrawn
    • Mount Sinai Hospital
  • Quebec
    • Montreal, Quebec, Canada
      • Recruiting
      • Sainte Justine Hospital
• Finland
  • Oulu, Finland, 90220
    • Recruiting
    • Oulun yliopistollinen sairaala
• France
  • Paris, France, 75015
    • Active, not recruiting
    • Groupe Hospitalier Necker Enfants Malades
  • Hauts-de-Seine
    • Clamart, Hauts-de-Seine, France, 92140
      • Active, not recruiting
      • Hôpital Antoine Béclère
• Germany
  • Nuremberg, Germany, 90479
    • Recruiting
    • Klinikum Nürnberg
  • Baden-Wurttemberg
    • Freiburg im Breisgau, Baden-Wurttemberg, Germany, 79106
      • Withdrawn
      • Universitätsklinikum Freiburg
  • Saxony
    • Leipzig, Saxony, Germany, 04103
      • Recruiting
      • Universitätsklinikum Leipzig
• Ireland
  • Wilton
    • Cork, Wilton, Ireland, T12YE02
      • Recruiting
      • Cork University Maternity Hospital
• Italy
  • Florence, Italy, 50134
    • Recruiting
    • Azienda Ospedaliero-Universitaria Careggi SOD Neonatologia e Terapia Intensiva Neonatale
  • Genova, Italy, 16147
    • Recruiting
    • Istituto Giannina Gaslini-Istituto Pediatrico di Ricovero e
  • Treviso, Italy, 31100
    • Recruiting
    • Presidio Ospedaliero Di Treviso Ca' Foncello
  • Lazio
    • Rome, Lazio, Italy, 00168
      • Recruiting
      • Fondazione Policlinico Universitario A Gemelli
  • Lombardy
    • Milan, Lombardy, Italy, 20122
      • Recruiting
      • Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
  • Veneto
    • Padua, Veneto, Italy, 35128
      • Recruiting
      • Azienda Ospedaliera Di Padova
• Japan
  • Tokyo, Japan, 183-8561
    • Recruiting
    • Tokyo Metropolitan Children's Medical Center
  • Tokyo, Japan, 142-8666
    • Recruiting
    • Showa Medical University Hospital
  • Kagoshima-ken
    • Kagoshima, Kagoshima-ken, Japan, 890-0055
      • Active, not recruiting
      • Kagoshima City Hospital
  • Nagano
    • Azumino, Nagano, Japan, 399-8205
      • Recruiting
      • Nagano Children's Hospital
  • Okayama-ken
    • Kurashiki-shi, Okayama-ken, Japan, 710-0052
      • Recruiting
      • Kurashiki Central Hospital
  • Saitama
    • Kawagoe-shi, Saitama, Japan, 350-8550
      • Recruiting
      • Saitama Medical Center
  • Ôsaka
    • Izumi, Ôsaka, Japan, 594-1101
      • Recruiting
      • Osaka Women's and Children's Hospital
• Netherlands
  • Utrecht, Netherlands, 3584 EA
    • Recruiting
    • Wilhelmina Children Hospital-University Medical Center Utrecht
  • Limburg
    • Maastricht, Limburg, Netherlands, 6229 HX
      • Recruiting
      • Maastricht University Medical Center
  • North Holland
    • Amsterdam-Zuidoost, North Holland, Netherlands, 1105 AZ
      • Recruiting
      • Academisch Medisch Centrum Amsterdam
• Portugal
  • Almada, Portugal, 2801-951
    • Recruiting
    • Hospital Garcia de Orta
  • Lisbon, Portugal, 1069-089
    • Recruiting
    • Maternidade Alfredo da Costa
  • Lisbon, Portugal, 1649-035
    • Recruiting
    • Centro Hospitalar Lisboa
  • Porto, Portugal, 4050-651
    • Recruiting
    • Centro Materno Infantil do Norte - Centro Hospital Universitario do Porto, E.P.E.
• Spain
  • Alicante, Spain, 03010
    • Recruiting
    • Hospital General Universitario Dr. Balmis
• Sweden
  • Lund, Sweden, SE-22185
    • Completed
    • Skanes universitetssjukhus
  • Stockholm, Sweden, 171 76
    • Withdrawn
    • Karolinska Solna
• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • Recruiting
    • University of Cambridge
  • Coventry, United Kingdom, CV2 2DX
    • Recruiting
    • University Hospital Coventry
  • Liverpool, United Kingdom, L69 3BX
    • Recruiting
    • Liverpool Women's Hospital - PPDS
  • London, United Kingdom, NW1 2BU
    • Withdrawn
    • University College London
  • London, United Kingdom, SW3 6JJ
    • Recruiting
    • Chelsea and Westminster NHS Trust
  • Manchester, United Kingdom, M13 9WL
    • Recruiting
    • St. Mary's Hospital
  • Norfolk
    • Norwich, Norfolk, United Kingdom, NR4 7UY
      • Recruiting
      • Norfolk and Norwich University Hospital
  • Surrey
    • Chertsey, Surrey, United Kingdom, KT16 0PZ
      • Completed
      • Ashford and St. Peter's Hospitals NHS Trust - St. Peter's Hospital
• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72202-3500
      • Recruiting
      • Arkansas Children's Hospital
    • Little Rock, Arkansas, United States, 72202-3500
      • Recruiting
      • University of Arkansas for Medical Sciences
  • California
    • California City, California, United States, 92868
      • Recruiting
      • Children's Hospital of Orange County
    • Los Angeles, California, United States, 90033-1804
      • Active, not recruiting
      • LAC USC Medical Center
  • Florida
    • Miami, Florida, United States, 33136-1005
      • Recruiting
      • Jackson Memorial Hospital
    • Tampa, Florida, United States, 33606-3571
      • Recruiting
      • Tampa General Hospital
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Recruiting
      • University of Illinois at Chicago
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Riley Hospital for Children
    • South Bend, Indiana, United States, 46601-1078
      • Recruiting
      • Memorial Hospital of South Bend
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Recruiting
      • Norton Children's Hospital
  • Louisiana
    • New Orleans, Louisiana, United States, 70115
      • Recruiting
      • Ochsner Baptist Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Withdrawn
      • Boston Children's Hospital
    • Boston, Massachusetts, United States, 02111-1553
      • Recruiting
      • Tufts Medical Center
  • Minnesota
    • Saint Paul, Minnesota, United States, 55102
      • Recruiting
      • Children's Minnesota - Children's Hospital and Clinics - St. Paul
    • Saint Paul, Minnesota, United States, 55102
      • Recruiting
      • Children's Minnesota - Children's Hospital and Clinics
  • Mississippi
    • Jackson, Mississippi, United States, 39216-4500
      • Recruiting
      • University of Mississippi Medical Center
  • New York
    • Rochester, New York, United States, 14627
      • Recruiting
      • University of Rochester
    • Valhalla, New York, United States, 10595
      • Recruiting
      • Maria Fareri Children's Hospital
  • Ohio
    • Columbus, Ohio, United States, 43205
      • Not yet recruiting
      • Nationwide Children's Hospital
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Recruiting
      • University of Oklahoma Health Sciences Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425-8908
      • Recruiting
      • Medical University of South Carolina Children Hospital
  • Virginia
    • Charlottesville, Virginia, United States, 22903
      • Recruiting
      • UVA Children's Hospital
    • Richmond, Virginia, United States, 23298-5075
      • Recruiting
      • Virginia Commonwealth University - Children's Hospital of Richmond at VCU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 second to 1 day (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. 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).
2. 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).
3. Subjects must be between 23 weeks +0 days and 27 weeks +6 days GA, inclusive.

Exclusion Criteria:

1. Detectable major (or severe) congenital malformation identified before randomization.
2. Known or suspected chromosomal abnormality, genetic disorder, or syndrome, identified before randomization, according to the investigator's opinion.
3. 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.
4. Clinically significant neurological disease identified before randomization according to cranial ultrasound (hemorrhages confined to the germinal matrix are allowed) and investigator's opinion.
5. 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.
6. 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).
7. 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.
8. Birth mother with active COVID-19 infection at birth or a history of severe COVID-19 infection (requiring intensive care hospitalization) during pregnancy.
9. Birth mother with known HIV or hepatitis (B, C, or E) infection.

Study Plan

How is the study designed?

Design Details

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

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Reduction in the incidence of severe Bronchopulmonary Dysplasia (BPD) at 36 weeks (±3 days) Postmenstrual Age (PMA), or death at or before 36 weeks PMA, 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
Incidence and severity of BPD	BPD severity is defined by the modified NICHD severity grading	Baseline through 36 weeks postmenstrual age (PMA)
Incidence of Retinopathy of Prematurity (ROP)	ROP is classified according to the International Classification	Baseline through 40 weeks PMA
Reducing the burden of Chronic Lung Disease, as indicated by a reduction in time to final weaning off of Respiratory Technology Support (RTS) through 12 months Corrected Age (CA), as compared to the SNC group.	The final weaning off of RTS is defined as the 7th consecutive day that the subject is off RTS.	Baseline through 12 months CA
Reduction in the incidence of severe BPD at 36 weeks (±3 days) PMA, or death at or before 36 weeks PMA, whichever comes first as compared to the SNC group.	Severe BPD is defined based on the classification according to Jensen et al., 2019	Time Frame: Baseline through 36 weeks postmenstrual age (PMA)
Occurrence of severe (Grade 3 and 4) intraventricular hemorrhage (IVH) before 40 weeks PMA, as assessed by cranial ultrasound as compared to the SNC group	Severe IVH as classified according to the Volpe criteria	Baseline through 40 weeks postmenstrual age (PMA)
To assess the effect of OHB-607 on occurrence of severe retinopathy of prematurity (ROP) (Stage 3 and above) up to 40 weeks PMA as compared to the SNC group		Baseline through 40 weeks postmenstrual age (PMA)
To assess the effect of OHB-607 on chronic respiratory outcomes as measured by the Chronic Lung Disease Prematurity Severity Score (CLDPSS) as compared to the SNC group at 12 months CA.		Baseline until 12 months CA using CLDPSS
The effect of OHB-607 on neurodevelopment is measured by the Cognitive, Language and Motor Scales of the Bayley Scales of Infant and Toddler Development (BSID) III as compared to the SNC group at 24 months CA.		Time Frame: Determined by the separate BSID III scales at 24 months CA
Chronic respiratory morbidity outcomes at 24 months CA		24 months CA
Jensen BPD grade at 36 weeks PMA (± 3 days), as classified according to Jensen et al., 2019. Incidence of all severity grades of BPD as assessed by Jensen et al., 2019		36 weeks weeks postmenstrual age (PMA) (± 3 days)
Incidence and severity of IVH	Incidence of all grades of IVH as assessed by centrally read CUS and 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 ASQ®-3 administered at 12 and 24 months CA.	From 6 months CA through 24 months CA
Mortality from randomization through to 24 months CA	Mortality rates from randomization to initial hospital discharge and from initial discharge through 24 months CA.	From birth through 24 months CA
Exposure-response relationship between measured IGF-1 and Bronchopulmonary Dysplasia (BPD)	Blood samples will be collected to measure IGF-1 and these measured values will be associated with the incidence and severity grade of BPD	Baseline through 36 weeks PMA
Exposure-response relationship between measured IGF-1 and intraventricular hemorrhage (IVH)	Blood samples will be collected to measure IGF-1 and these measured values will be associated with the incidence and severity grade of IVH	Baseline through 40 weeks PMA
Exposure-response relationship between measured IGF-1 and necrotizing enterocolitis (NEC)	Blood samples will be collected to measure IGF-1 and these measured values will be associated with the incidence and severity grade of NEC	Baseline through 40 weeks PMA
Exposure-response relationship between measured IGF-1 and Retinopathy of Prematurity (ROP)	Blood samples will be collected to measure IGF-1 and these measured values will be associated with the incidence and severity grade of ROP	Baseline through 40 weeks PMA
To assess the safety profile of OHB-607 as compared to the SNC group.	Incidence, severity, and causality assessment of Adverse Events (AEs) and Serious Adverse Events (SAEs), including Fatal AEs as per the neonatal adverse event severity scale.	Baseline through 24 months CA

Collaborators and Investigators

• Sponsor: OHB Neonatology Ltd.

Publications and helpful links

General Publications

• Kramer BW, Abman S, Daly M, Jobe AH, Niklas V. Insulin-like growth factor-1 replacement therapy after extremely premature birth: An opportunity to optimize lifelong lung health by preserving the natural sequence of lung development. Paediatr Respir Rev. 2023 Dec;48:24-29. doi: 10.1016/j.prrv.2023.05.001. Epub 2023 May 6.
• Ley D, Hallberg B, Hansen-Pupp I, Dani C, Ramenghi LA, Marlow N, Beardsall K, Bhatti F, Dunger D, Higginson JD, Mahaveer A, Mezu-Ndubuisi OJ, Reynolds P, Giannantonio C, van Weissenbruch M, Barton N, Tocoian A, Hamdani M, Jochim E, Mangili A, Chung JK, Turner MA, Smith LEH, Hellstrom A; study team. rhIGF-1/rhIGFBP-3 in Preterm Infants: A Phase 2 Randomized Controlled Trial. J Pediatr. 2019 Mar;206:56-65.e8. doi: 10.1016/j.jpeds.2018.10.033. Epub 2018 Nov 22.
• Hellstrom W, Hortensius LM, Lofqvist C, Hellgren G, Tataranno ML, Ley D, Benders MJNL, Hellstrom A, Bjorkman-Burtscher IM, Heckemann RA, Savman K. Postnatal serum IGF-1 levels associate with brain volumes at term in extremely preterm infants. Pediatr Res. 2023 Feb;93(3):666-674. doi: 10.1038/s41390-022-02134-4. Epub 2022 Jun 9.

Study record dates

Study Major Dates

• Study Start (Actual): May 9, 2019
• Primary Completion (Estimated): January 23, 2026
• Study Completion (Estimated): January 21, 2028

Study Registration Dates

• First Submitted: August 15, 2017
• First Submitted That Met QC Criteria: August 15, 2017
• First Posted (Actual): August 17, 2017

Study Record Updates

• Last Update Posted (Estimated): January 7, 2026
• Last Update Submitted That Met QC Criteria: January 5, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Eye Diseases; Retinal Diseases; Lung Injury; Ventilator-Induced Lung Injury; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Retinopathy of Prematurity; Bronchopulmonary Dysplasia; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors; Blood Proteins; Somatomedins; Insulin-Like Peptides; Intercellular Signaling Peptides and Proteins; Insulin-Like Growth Factor I
• Other Study ID Numbers: OHB-607-202; 2018-001393-16 (EudraCT Number); jRCT2071200076 (Registry Identifier: jRCT)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: De-identified individual participant data from this particular study will not be shared in order to minimize the risk that individual patients could be re-identified, given that there are limited numbers of study participants at each study site per year.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No