Clinical Trials Register

Summary
EudraCT Number:	2018-001393-16
Sponsor's Protocol Code Number:	OHB-607-202
National Competent Authority:	Portugal - INFARMED
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2019-02-19
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Portugal - INFARMED

A.2

EudraCT number

2018-001393-16

A.3

Full title of the trial

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

Um estudo de Fase 2b, multicêntrico, aleatorizado, em regime aberto, de dois braços para avaliar a eficácia clínica e a segurança do OHB-607 em comparação com os cuidados neonatais habituais na prevenção da displasia broncopulmonar, a causa mais comum de doença pulmonar crónica da prematuridade

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A study evaluating the safety and efficacy of an experimental drug for chronic lung disease against normally prescribed care in extremely premature babies

A.4.1

Sponsor's protocol code number

OHB-607-202

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03253263

A.5.4

Other Identifiers

Name:

IND number

Number:

133076

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/066/2020

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	OHB Neonatology Ltd
B.1.3.4	Country	United Kingdom
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	OHB Neonatology Ltd
B.4.2	Country	United Kingdom
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	OHB Neonatology Ltd
B.5.2	Functional name of contact point	Customer Services
B.5.3	Address:
B.5.3.1	Street Address	1 Ashley Road, 3rd Floor
B.5.3.2	Town/ city	Altrincham, Cheshire
B.5.3.3	Post code	WA14 2DT
B.5.3.4	Country	United Kingdom
B.5.6	E-mail	info@oakhillbio.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/06/399
D.3 Description of the IMP
D.3.1	Product name	OHB-607 (mecasermin rinfabate, rhIGF-1/rhIGFBP-3)
D.3.2	Product code	OHB-607
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Mecasermin rinfabate
D.3.9.1	CAS number	478166-15-3
D.3.9.2	Current sponsor code	OHB-607, HGT-ROP001
D.3.10	Strength
D.3.10.1	Concentration unit	µg/ml microgram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Chronic Lung Disease

E.1.1.1

Medical condition in easily understood language

Chronic Lung Disease

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	LLT
E.1.2	Classification code	10066204
E.1.2	Term	Chronic lung disease of prematurity
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the effect of OHB-607 on reducing the burden of CLD, as
indicated by a reduction in the incidence of severe BPD (as defined by
the modified NICHD severity grading) at 36 weeks (±3 days) PMA, or
death at or before 36 weeks PMA, whichever comes first as compared to
the SNC group.

E.2.2

Secondary objectives of the trial

•To assess the effect of OHB-607 on reducing the burden of CLD, as
indicated by a reduction in time to final weaning off of RTS through 12
months CA, as compared to the SNC group
•To assess the effect of OHB-607 on reducing the burden of CLD, as
indicated by a reduction in the incidence of Grade 2 and Grade 3 (severe)
BPD at 36 weeks (±3 days) PMA, or death, whichever comes first as
compared to the SNC group, as classified according to Jensen et al., 2019
•To assess the effect of OHB-607 on the occurrence of severe (Grade 3
and 4) IVH before 40 weeks PMA, as assessed by CUS as compared to
the SNC group
•To assess the effect of OHB-607 on occurrence of severe ROP (Stage 3
and above) up to 40 weeks PMA as compared to the SNC group
•To assess the effect of OHB-607 on chronic respiratory outcomes as
measured by the CLDPSS as compared to the SNC group at 12 months CA
Refer to protocol for full list of secondary objectives

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consents and/or assents must be signed and dated by the subject'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 subject's birth mother prior to providing study-related information related to birth mother medical history, pregnancy and the birth of the subject. 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.

E.4

Principal 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 <45 mg/dL or 2.5 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 subject or interfere with the subject'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 subject or subject'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.
Major (or severe) congenital malformations include structurally significant congenital heart disease, and structural abnormalities of the upper airway, lungs or chest wall. Congenital malformations that are suspected of being associated with chromosomal abnormalities, genetic syndromes, and neoplasia should be excluded, as well as abnormalities that may affect life expectancy, cardiopulmonary development, neurologic development, or
interpretation of study results.
Isolated minor dysmorphic anomalies that are unlikely to be exclusionary could include post-axial polydactyly, ankyloglossia, accessory nipples, preauricular pits, single or horizontal palmar crease,
clinodactyly, and single umbilical artery. However, the presence of multiple minor anomalies in the same infant may be exclusionary.
Uncomplicated infantile hemangiomas are unlikely to be exclusionary.
However, subjects with infantile hemangiomas that may be associated with potential for disfigurement, life-threatening complications, functional impairment, ulceration, or underlying abnormalities should be excluded.
Inclusion/exclusion will ultimately be determined by the investigator, based on assessment of the clinical presentation of each candidate subject and the likelihood that physical finding(s) are associated with a condition that impacts health and development.

E.5 End points

E.5.1

Primary end point(s)

Incidence of severe BPD (as defined by the modified NICHD severity
grading) or death for all subjects at or before 36 weeks (±3 days) PMA.
The definitions for BPD are based upon the modified NICHD guidelines
for preterm infants born at <32 weeks GA:
- No BPD: oxygen for <28 days or none.
- Mild BPD: a need for oxygen for ≥28 days but on room air at 36 weeks
PMA.
- Moderate BPD: oxygen for ≥28 days plus treatment with <30% oxygen
at 36 weeks PMA.
- Severe BPD: oxygen for ≥28 days plus oxygen ≥30% and/or any
positive pressure ventilation (CPAP, IMV, NNIMV, or high flow nasal
cannula ≥2 L/min) at 36 weeks PMA.

E.5.1.1

Timepoint(s) of evaluation of this end point

At 36 weeks PMA.

E.5.2

Secondary end point(s)

• Time to final weaning off of RTS from Day 1 of randomization through
12 months CA. The final weaning off of RTS is defined as the 7th
consecutive day that the subject is off RTS.
• Incidence of Grade 2 and Grade 3 (severe) BPD (as defined by the
modified Jensen severity grading) or death for all subjects at 36 weeks
PMA. The definitions for BPD are based on the classification according to
Jensen et al., 2019:
- No BPD: no support.
- Grade 1: supplemental oxygen <2 L/min without positive pressure
(including nasal cannula).
- Grade 2: positive pressure support (including CPAP, nasal cannula
oxygen =2 L/min, NIPPV).
- Grade 3: positive pressure ventilation (high-frequency oscillation
ventilation and technologies with positive pressure tidal volume breaths,
such as IMV).
• Incidence of severe (Grade 3 and 4) IVH before 40 weeks PMA (or
discharge from/transfer from the NICU, whichever comes first) as
assessed by central blinded reviewers and classified according to the
Volpe:
- Grade 1: blood in the germinal matrix with or without IVH <10% of
ventricular space.
- Grade 2: IVH occupying 10 to 50% of ventricular space on parasagittal
view.
- Grade 3: IVH occupying >50% of ventricle with or without
periventricular echo densities.
- Grade 4: evidence of posthemorrhagic infarction or periventricular echo
densities.
• Incidence of severe ROP (Stage 3 and above) up to 40 weeks PMA
according to International Classification (International Committee for
the Classification of Retinopathy of Prematurity, 2021) by local blinded
reviewer.
• Respiratory severity scoring will be determined from information
captured during follow-up telephone calls and clinical site visits at
intervals specified until 12 months CA using CLDPSS.
• Neurodevelopmental impairment as determined by the separate BSID
III scales at 24 months CA.
- Motor composite score
- Cognitive composite score
- Language composite score
Refer protocol for other Secondary end points

E.5.2.1

Timepoint(s) of evaluation of this end point

At 40 weeks PMA and at 12 and 24 months CA.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Standard of care

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

Japan

United Kingdom

United States

Finland

France

Germany

Ireland

Italy

Netherlands

Portugal

Spain

Sweden

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The date the final subject, across all sites, completes their final
protocol-defined assessment

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

338

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

Yes

F.1.1.2.1

Number of subjects for this age range:

338

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

The participants will be infants born prematurely. Consent will be sought from the parents or legal guardian.

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

338

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

After trial completion, subjects will return to standard of care for any ongoing health issues.

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2019-05-06

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-09-02

P. End of Trial
P.	End of Trial Status	Trial now transitioned

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