Bronchopulmonary DysplasiaPhase 2NCT03253263
A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants
A Phase 2 study testing OHB-607 in people with Bronchopulmonary Dysplasia and Chronic Lung Disease of Prematurity, run by OHB Neonatology Ltd. at 62 sites in 13 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 0 to 24 hours
- Conditions the study is for
- Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage
- What is being tested?
- OHB-607
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 295 participants
- Where
- 62 sitesin 13 countries
- Study dates
- 9 May 2019to 21 Jan 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 to 24 hours
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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).
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- 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.
- Birth mother with known HIV or hepatitis (B, C, or E) infection.
Trial locations and contacts
62 sites in 13 countries.
62 sites in 13 countries
United States24 sites
Tufts Medical Center
Boston, Massachusetts, 02111-1553
Children's Hospital of Orange County
California City, California, 92868
Medical University of South Carolina Children Hospital
Charleston, South Carolina, 29425-8908
UVA Children's Hospital
Charlottesville, Virginia, 22903
University of Illinois at Chicago
Chicago, Illinois, 60612
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
University of Mississippi Medical Center
Jackson, Mississippi, 39216-4500
Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3500
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72202-3500
Cedars Sinai Medical Center
Los Angeles, California, 90048
University of Louisville Hospital
Louisville, Kentucky, 40202
Jackson Memorial Hospital
Miami, Florida, 33136-1005
Ochsner Baptist Medical Center
New Orleans, Louisiana, 70115
Oklahoma Children's Hospital - PIN
Oklahoma City, Oklahoma, 73104
Virginia Commonwealth University - Children's Hospital of Richmond at VCU
Richmond, Virginia, 23298-5075
University of Rochester
Rochester, New York, 14627
US Davis
Sacramento, California, 95817
Children's Minnesota - Children's Hospital and Clinics - St. Paul
Saint Paul, Minnesota, 55102
Children's Minnesota - Children's Hospital and Clinics
Saint Paul, Minnesota, 55102
UT Health Science Center
San Antonio, Texas, 78229
Memorial Hospital of South Bend
South Bend, Indiana, 46601-1078
Tampa General Hospital
Tampa, Florida, 33606-3571
Maria Fareri Children's Hospital
Valhalla, New York, 10595
Japan8 sites
Nagano Children's Hospital
Azumino, Nagano, 399-8205
Osaka Women's and Children's Hospital
Izumi, Ôsaka, 594-1101
Saitama Medical Center
Kawagoe-shi, Saitama, 350-8550
Kurashiki Central Hospital
Kurashiki-shi, Okayama-ken, 710-0052
Saitama Prefectural Children's Medical Center
Saitama, Saitama, 330-8777
Showa Medical University Hospital
Tokyo, 142-8666
National Center for Child Health and Development
Tokyo, 157-8535
Tokyo Metropolitan Children's Medical Center
Tokyo, 183-8561
United Kingdom7 sites
University of Cambridge
Cambridge, CB2 0QQ
Ashford and St. Peter's Hospitals NHS Trust - St. Peter's Hospital
Chertsey, Surrey, KT16 0PZ
University Hospital Coventry
Coventry, CV2 2DX
Liverpool Women's Hospital - PPDS
Liverpool, L69 3BX
Chelsea and Westminster NHS Trust
London, SW3 6JJ
St. Mary's Hospital
Manchester, M13 9WL
Norfolk and Norwich University Hospital
Norwich, Norfolk, NR4 7UY
Italy5 sites
Azienda Ospedaliero-Universitaria Careggi SOD Neonatologia e Terapia Intensiva Neonatale
Florence, 50134
Istituto Giannina Gaslini-Istituto Pediatrico di Ricovero e
Genova, 16147
Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
Milan, Lombardy, 20122
Azienda Ospedaliera Di Padova
Padova, Veneto, 35128
Presidio Ospedaliero Di Treviso Ca' Foncello
Treviso, 31100
Australia4 sites
Royal Women's Hospital
Parkville, 3052
Royal Hospital for Women
Randwick, New South Wales, 2031
Mater Misericordiae Limited
South Brisbane, Qld 4101
Westmead Hospital
Westmead, New South Wales, NSW 2145
Portugal4 sites
Hospital Garcia de Orta
Almada, 2801-951
Maternidade Alfredo da Costa
Lisbon, 1069-089
Centro Hospitalar Lisboa
Lisbon, 1649-035
Centro Materno Infantil do Norte - Centro Hospital Universitario do Porto, E.P.E.
Porto, 4050-651
Netherlands3 sites
Academisch Medisch Centrum Amsterdam
Amsterdam-Zuidoost, North Holland, 1105 AZ
Maastricht University Medical Center
Maastricht, Limburg, 6229 HX
Wilhelmina Children Hospital-University Medical Center Utrecht
Utrecht, 3584 EA
Germany2 sites
Universitatsklinikum Leipzig
Leipzig, Saxony, 04103
Klinikum Nürnberg
Nuremberg, 90479
About this study
Official title: 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.
- Conditions
- Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity, Intraventricular Hemorrhage, Retinopathy of Prematurity (ROP)
- Being tested
- OHB-607
- Medical subject headings
- Bronchopulmonary Dysplasia, Retinopathy of Prematurity
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OHB-607Drug
Also called Mecasermin Rinfabate
Participants will receive intravenous infusion of OHB-607 from birth up to PMA 29 weeks + 6 days.
| Study group | Role | What they receive |
|---|---|---|
| OHB-607 | Experimental | Participants will receive continuous IV infusion of OHB-607 through from birth up to PMA 29 weeks +6 days. Receives: Drug: OHB-607 |
| Standard Neonatal Care | No intervention | Standard neonatal care alone will be provided. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Baseline through 36 weeks postmenstrual age (PMA)
Secondary outcomes (18)
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.
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: Time Frame: Determined by the separate BSID III scales at 24 months CA
Chronic respiratory morbidity outcomes at 24 months CA
Measured: 24 months CA
Incidence and severity of BPD
BPD severity is defined by the modified NICHD severity grading
Measured: Baseline through 36 weeks postmenstrual age (PMA)
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
Measured: 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
Measured: 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.
Measured: From 6 months CA through 24 months CA
Incidence of Retinopathy of Prematurity (ROP)
ROP is classified according to the International Classification
Measured: Baseline through 40 weeks PMA
Mortality from randomization through to 24 months CA
Mortality rates from randomization to initial hospital discharge and from initial discharge through 24 months CA.
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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.
Measured: Baseline through 24 months CA
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Aug 2017
- Study start9 May 2019
- Primary completion31 Jul 2026 (estimated)
- Study completion21 Jan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Bronchopulmonary DysplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 295 participants (actual)
Sponsor and investigators
- Lead sponsor
- OHB Neonatology Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03253263
- Sponsor's protocol ID
- OHB-607-202
- Other IDs
- 2018-001393-16, jRCT2071200076, 2018
- Status last verified
- Jul 2026
- First submitted
- 15 Aug 2017
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: 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.
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.
PubMed 37268507Ley 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.
PubMed 30471715Baraldi E, De Luca D, Bonadies L, Hirano S, Kusuda S, Bancalari E, Ramanathan R, Barton N, Nickless A, Lee J, Mahajan N, Niklas V. Randomised al.Phase 2b trial of rhIGF-1/rhIGFBP-3 (OHB-607) for bronchopulmonary dysplasia prevention in preterm neonates: study protocol. BMJ Paediatr Open. 2026 Mar 5;10(1):e004196. doi: 10.1136/bmjpo-2025-004196.
PubMed 41786363Hellstrom 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.
PubMed 35681088