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Summary
EudraCT Number:	2013-005429-21
Sponsor's Protocol Code Number:	D20496
National Competent Authority:	Slovenia - JAZMP
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2015-09-02
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

A. Protocol Information

A.1

Member State Concerned

Slovenia - JAZMP

A.2

EudraCT number

2013-005429-21

A.3

Full title of the trial

Multicentre randomised controlled trials of minimally-invasive surfactant
therapy in preterm infants 25 – 28 weeks gestation on continuous
positive airway pressure.

A.3.1

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

Premature infants between 25 and 28 weeks gestation may develop lung
disease and require respiratory support of continuous positive airway
pressure (CPAP). The trial is looking at if there is a benefit from giving a
medication called surfactant at an early stage, delivered via a narrow
tube briefly placed into the windpipe.

A.4.1

Sponsor's protocol code number

D20496

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02140580

A.5.4

Other Identifiers

Name:

Australia New Zealand Clinical Trial Registry

Number:

12611000916943

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Menzies Institute of Medical Research, University of Tasmania
B.1.3.4	Country	Australia
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	National Health and Medical Research Council
B.4.2	Country	Australia
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Royal Hobart Hospital
B.5.2	Functional name of contact point	Prof Peter Dargaville
B.5.3	Address:
B.5.3.1	Street Address	Liverpool St
B.5.3.2	Town/ city	Hobart
B.5.3.3	Post code	7000
B.5.3.4	Country	Australia
B.5.6	E-mail	peter.dargaville@dhhs.tas.gov.au

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Comparator
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	Yes
D.2.1.1.1	Trade name	Curosurf
D.2.1.1.2	Name of the Marketing Authorisation holder	Chiesi Slovenija d.o.o.
D.2.1.2	Country which granted the Marketing Authorisation	Slovenia
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Curosurf
D.3.4	Pharmaceutical form	Endotracheopulmonary instillation, solution
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP
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)	No
	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

Respiratory Distress Syndome in Premature Babies

E.1.1.1

Medical condition in easily understood language

Premature baby immature lung disease.

E.1.1.2

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

MedDRA Classification

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 evaluate in a randomised controlled trial the efficacy of surfactant
delivery via a minimally invasive technique in preterm infants 25-28
weeks gestation with RDS treated with CPAP.

E.2.2

Secondary objectives of the trial

To evaluate that early surfactant administration via a minimallyinvasive
technique to preterm infants on CPAP will result in a lesser
duration of mechanical respiratory support, and a higher incidence of
survival without bronchopulmonary dysplasia.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Requiring CPAP or nasal IPPV because of respiratory distress.
2. CPAP pressure of 5-8 cm H2O and FiO2 ≥0.30.
3. Less than 6 hours of age.
4. Agreement of the Treating Physician in charge of the infant's care.
5. Signed parental consent.

E.4

Principal exclusion criteria

1. Previously intubated, or in imminent need of intubation because of
respiratory distress, apnoea or persistent acidosis.
2. Congenital anomaly or condition that might adversely affect
breathing.
3. Identifiable alternative cause for respiratory distress (e.g. congenital
pneumonia or pulmonary hypoplasia).
4. Lack of availability of an OPTIMIST treatment team.

E.5 End points

E.5.1

Primary end point(s)

Incidence of composite outcome of death or physiological BPD

E.5.1.1

Timepoint(s) of evaluation of this end point

36 weeks gestational age

E.5.2

Secondary end point(s)

• Physiological BPD
• Clinical BPD (requirement for oxygen or any form of positive
pressure support at 36
weeks corrected gestation).
• Mild/moderate/severe BPD
• Death
• Death or BPD (clinical definition)
• Intraventricular haemorrhage (IVH) (all grades)
• IVH grades III and IV
• Periventricular leukomalacia
• Retinopathy of prematurity > stage II
• Major morbidity (any of IVH grade III or IV, periventricular
leukomalacia, ROP >stage II, physiological BPD)
• Death or major morbidity
• NEC (Modified Bell stage 2 or greater)
• NEC or spontaneous intestinal perforation requiring surgery
• Requirement for intubation in the first 72 hours of life
• Requirement for intubation at any time
• Need for additional surfactant therapy
• Overall number of surfactant doses (including that given by MIST)
• Duration of intubation (all episodes)
• Duration of CPAP/NIPPV (all episodes)
• Duration of intubation and CPAP
• Duration of high flow nasal cannula (HFNC), minimum flow rate 2
L/min
• Duration of respiratory support
• Duration of oxygen therapy
• Requirement for oxygen at home
• Length of stay in intensive care
• Length of hospital stay
• Total hospital billings
• Calculated cost of hospitalisation
• Pneumothorax requiring drainage
• Pulmonary haemorrhage
• Patent ductus arteriosus (PDA) requiring anti-prostaglandin therapy
• PDA requiring ligation
• Late onset sepsis (positive bacterial or fungal culture from a
normally sterile site)
• Time to regain birth weight
• Incidence of successful surfactant administration via MIST
• Number of catheterisation attempts
• Duration of bradycardia and hypoxaemia
• Requirement for, and duration of, positive pressure ventilation by
mask
• Incidence of apparent discomfort

E.5.2.1

Timepoint(s) of evaluation of this end point

Sischarge of baby from the hospital.

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

Yes

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

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)

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

Yes

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

A sham procedure will be performed.

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

A sham procedure will be performed.

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

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

LVLS

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

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:

606

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:

606

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

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

25-28 week gestation babies

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.4.2.2

In the whole clinical trial

606

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)

Normal standard treatment.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	University Medical Centre Ljubljana
G.4.3.4	Network Country	Slovenia

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2015-11-10

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2014-11-17

P. End of Trial
P.	End of Trial Status	Ongoing

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