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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Intraventricular Hemorrhage; Bronchopulmonary Dysplasia; Chronic Lung Disease of Prematurity; Retinopathy of Prematurity (ROP)
• Intervention / Treatment: Drug: OHB-607

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

Contacts and Locations

Study Locations

• Canada
  • Toronto, Canada, M5G 1X5
    • Mount Sinai Hospital
  • Quebec
    • Montreal, Quebec, Canada
      • Sainte Justine Hospital
• Finland
  • Oulu, Finland, 90220
    • Oulun yliopistollinen sairaala
• France
  • Paris, France, 75015
    • Groupe Hospitalier Necker Enfants Malades
  • Hauts-de-Seine
    • Clamart, Hauts-de-Seine, France, 92140
      • Hôpital Antoine Béclère
• Germany
  • Nürnberg, Germany, 90479
    • Klinikum Nürnberg
  • Baden-Württemberg
    • Freiburg, Baden-Württemberg, Germany, 79106
      • Universitätsklinikum Freiburg
  • Sachsen
    • Leipzig, Sachsen, Germany, 04103
      • Universitätsklinikum Leipzig
• Ireland
  • Wilton
    • Cork, Wilton, Ireland
      • Cork University Maternity Hospital
• Italy
  • Firenze, Italy, 50134
    • Azienda Ospedaliero-Universitaria Careggi SOD Neonatologia e Terapia Intensiva Neonatale
  • Genova, Italy, 16147
    • Istituto Giannina Gaslini-Istituto Pediatrico di Ricovero e
  • Treviso, Italy, 31100
    • Presidio Ospedaliero Di Treviso Ca' Foncello
  • Lazio
    • Roma, Lazio, Italy, 00168
      • Fondazione Policlinico Universitario A Gemelli
  • Lombardia
    • Milano, Lombardia, Italy, 20122
      • Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
  • Veneto
    • Padova, Veneto, Italy, 35128
      • Azienda Ospedaliera di Padova
• Japan
  • Kagosima
    • Kagoshima-shi, Kagosima, Japan, 890-0075
      • Kagoshima City Hospital
  • Nagano
    • Azumino, Nagano, Japan, 399-8205
      • Nagano Children's Hospital
  • Okayama
    • Kurashiki-shi, Okayama, Japan, 710-0052
      • Kurashiki Central Hospital
  • Saitama
    • Kawagoe-shi, Saitama, Japan, 350-8550
      • Saitama Medical Center
  • Ôsaka
    • Izumi, Ôsaka, Japan, 594-1101
      • Osaka Women's and Children's Hospital
• Netherlands
  • Utrecht, Netherlands, 3584 EA
    • Wilhelmina Children Hospital-University Medical Center Utrecht
  • Limburg
    • Maastricht, Limburg, Netherlands, 6229 HX
      • Maastricht University Medical Center
  • Noord-Holland
    • Amsterdam-Zuidoost, Noord-Holland, Netherlands, 1105 AZ
      • Academisch Medisch Centrum Amsterdam
• Portugal
  • Almada, Portugal, 2801-951
    • Hospital Garcia de Orta
  • Lisboa, Portugal, 1069-089
    • Maternidade Alfredo da Costa
  • Lisboa, Portugal, 1649-035
    • Centro Hospitalar Lisboa
  • Porto, Portugal, 4050-651
    • Centro Materno Infantil do Norte - Centro Hospital Universitario do Porto, E.P.E.
• Spain
  • Alicante, Spain, 03010
    • Hospital General Universitario Dr. Balmis
• Sweden
  • Lund, Sweden, SE-22185
    • Skanes universitetssjukhus
  • Stockholm, Sweden, 171 76
    • Karolinska Solna
• United Kingdom
  • Cambridge, United Kingdom, CB2 0QQ
    • University of Cambridge
  • London, United Kingdom, NW1 2BU
    • University College London
  • London, United Kingdom, SW3 6JJ
    • Chelsea and Westminster NHS Trust
  • Manchester, United Kingdom, M13 9WL
    • St. Mary's Hospital
  • Norfolk
    • Norwich, Norfolk, United Kingdom, NR4 7UY
      • Norfolk and Norwich University Hospital
  • Surrey
    • Chertsey, Surrey, United Kingdom, KT16 0PZ
      • Ashford and St. Peter's Hospitals NHS Trust - St. Peter's Hospital
• United States
  • Alabama
    • Mobile, Alabama, United States, 36604
      • University of South Alabama Children's and Women's Hospital
  • Arkansas
    • Little Rock, Arkansas, United States, 72202-3500
      • Arkansas Children's Hospital
    • Little Rock, Arkansas, United States, 72202-3500
      • University of Arkansas for Medical Sciences
  • California
    • Los Angeles, California, United States, 90033
      • LAC USC Medical Center
  • Florida
    • Tampa, Florida, United States, 33606
      • Tampa General Hospital
  • Illinois
    • Chicago, Illinois, United States, 60612
      • University of Illinois at Chicago
  • Indiana
    • South Bend, Indiana, United States, 46601
      • Memorial Hospital of South Bend
  • Louisiana
    • New Orleans, Louisiana, United States, 70115
      • Ochsner Baptist Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02111
      • Floating Hospital for Children
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
  • Mississippi
    • Jackson, Mississippi, United States, 39216
      • University of Mississippi Medical Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma Health Sciences Center
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina Children Hospital
  • Virginia
    • Richmond, Virginia, United States, 23219
      • 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
• Genders Eligible for Study: All

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.

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 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

Collaborators and Investigators

• Sponsor: OHB Neonatology Ltd.

Publications and helpful links

General Publications

• 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. 2022 Jun 9. doi: 10.1038/s41390-022-02134-4. Online ahead of print.

Study record dates

Study Major Dates

• Study Start (Actual): May 9, 2019
• Primary Completion (Anticipated): August 28, 2026
• Study Completion (Anticipated): November 28, 2026

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 (Estimate): December 12, 2022
• Last Update Submitted That Met QC Criteria: December 9, 2022
• Last Verified: December 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Respiratory Tract Diseases; Eye Diseases; Infant, Newborn, Diseases; Retinal Diseases; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Lung Injury; Infant, Premature, Diseases; Ventilator-Induced Lung Injury; Lung Diseases; Hemorrhage; Premature Birth; Retinopathy of Prematurity; Bronchopulmonary Dysplasia; Physiological Effects of Drugs; Growth Substances; Mecasermin
• 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