Clinical Trials Register

Summary
EudraCT Number:	2015-003519-40
Sponsor's Protocol Code Number:	03-CL-1202
National Competent Authority:	Poland - Office for Medicinal Products
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2016-01-07
Trial results	View 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

Poland - Office for Medicinal Products

A.2

EudraCT number

2015-003519-40

A.3

Full title of the trial

A MULTINATIONAL, MULTICENTER, MASKED, RANDOMIZED, CONTROLLED
STUDY TO ASSESS THE SAFETY AND EFFICACY OF LUCINACTANT FOR
INHALATION IN PRETERM NEONATES 26 TO 32 WEEKS GESTATIONAL AGE
WITH RESPIRATORY DISTRESS SYNDROME.

A.3.1

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

An international study at differnet study sites testing the safety and effectiveness of a drug, lucinactant, which is to be inhaled as a means to treat breathing problems in babies born prematurely in the 26 to 32 week

A.4.1

Sponsor's protocol code number

03-CL-1202

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT02636868

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	Windtree Therapeutics, Inc
B.1.3.4	Country	United States
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	Windtree Therapeutics, Inc
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Clinical Consulting Sp. z o.o.
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	Dzwonkowa 104
B.5.3.2	Town/ city	Tychy
B.5.3.3	Post code	43-100
B.5.3.4	Country	Poland
B.5.4	Telephone number	+48322272005
B.5.5	Fax number	+48323298426
B.5.6	E-mail	office@clinicalconsulting.pl

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/3/04/216; EU/3/04/217
D.3 Description of the IMP
D.3.1	Product name	Lyophilized lucinactant
D.3.4	Pharmaceutical form	Nebuliser suspension
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Respiratory use (Noncurrent)
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	dipalmitoylphosphatidylcholine
D.3.9.1	CAS number	63-89-8
D.3.9.3	Other descriptive name	1,2-DIPALMITOYL-SN-GLYCERO-3-PHOSPHATIDYLCHOLINE (DPPC)
D.3.9.4	EV Substance Code	SUB22681
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	22,5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Palmitic acid
D.3.9.1	CAS number	57-10-3
D.3.9.3	Other descriptive name	PALMITIC ACID
D.3.9.4	EV Substance Code	SUB14744MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	4,05
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	palmitoyloleoyl-phosphatidylglycerol
D.3.9.1	CAS number	268550-95-4
D.3.9.3	Other descriptive name	PALMITOYL-OLEOYL PHOSPHATIDYLGLYCEROL
D.3.9.4	EV Substance Code	SUB126307
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	7,5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	SINAPULTIDE
D.3.9.1	CAS number	138531-07-4
D.3.9.4	EV Substance Code	SUB10530MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	0,862
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
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	Yes
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 Syndrome (RDS)

E.1.1.1

Medical condition in easily understood language

Breathing difficulty in babies born prematurely

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	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10021735
E.1.2	Term	Infant respiratory distress syndrome
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10038690
E.1.2	Term	Respiratory distress syndrome (neonatal)
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10028974
E.1.2	Term	Neonatal respiratory distress syndrome
E.1.2	System Organ Class	10038738 - Respiratory, thoracic and mediastinal disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the safety and efficacy of lucinactant for inhalation in conjuction with nasal continuous positive airway pressure (nCPAP), in comparison to nCPAP alone, in preterm neonates with RDS, as assessed by the time to, and incidence of, respiratory failure and/or death due to RDS, incidence of bronchopulmonary dysplasia (BPD) at 36 weeks postmenstrual age (PMA), and change in physiologic parameters (fraction of inspired oxygen [FiO2] and partial pressure of carbon dioxide [PCO2]) over the first 72 hours of life.

E.2.2

Secondary objectives of the trial

Not applicable

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Signed ICF from legally authorized representative
2. 26 0/7 to 32 6/7 completed weeks' gestation PMA
3. Successful implementation of non-invasive support or ventilation
within 90 minutes after birth
4. Spontaneous breathing
5. Chest radiograph consistent with RDS
6. Within the first 20 hours after birth, requires an nCPAP of 5 to 7 cm H2O with an FiO2 ≥0.25 (>0.21 for neonates 26-28 weeks PMA) to 0.4 that is clinically indicated for at least 30 minutes to maintain SpO2 of 90% to 95%. Transient (<10 minutes) FiO2 excursions below 0.25 or above 0.45 do not reset the 30-minute requirement.

E.4

Principal exclusion criteria

1. A heart rate that cannot be stabilized above 100 beats per minute
(bpm) within 5 minutes of birth
2. Recurrent episodes of apnea requiring positive pressure ventilation (PPV) administered manually or mechanically through any patient interface
3. A 5 minute Apgar score < 5
4. Major congenital malformation(s) or craniofacial abnormalities that preclude the use of nCPAP, diagnosed antenatally or immediately after birth
5. Clinically significant diseases or conditions other than RDS which could potentially interfere with cardiopulmonary function (eg, congenital heart disease, hydrops fetalis or congenital infection)
6. A known or suspected chromosomal abnormality or syndrome
7. Premature rupture of membranes (PROM) >3 weeks
8. Hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis
9. A need for intubation and/or invasive mechanical ventilation at any
time before enrollment into the study
10. The administration (or plan for administration) of any the following:
a) Another investigational agent or investigational medical device
b) Any other surfactant agent
c) Systemic corticosteroids (other than antenatal steroids already received)
11. Presence of air leak (pneumothorax, pneumomediastinum, pneumopericardium, subcutaneous emphysema, or definite evidence of pulmonary interstitial emphysema [PIE]) on the baseline chest
radiograph

E.5 End points

E.5.1

Primary end point(s)

The primary endpoint for this study is time to respiratory failure or death due to RDS within the first 72 hours of life.
Respiratory failure due to RDS will be defined as follows:
a. any subject receiving intubation for MV and/or surfactant administration within 72 hours of life.
b. if at least 1 of the following criteria are met, regardless of whether endotracheal intubation is performed:
• a sustained (≥ 60 minutes) need for FiO2 > 0.45 to maintain an SpO2 > 90% to 95%.
• a sustained PCO2 > 65 mm Hg on ≥ 2 consecutive observations (> 60 minutes apart) transcutaneous PCO2 > 65 mm Hg
• nCPAP ≥ 8 cm H2O
Death due to RDS is any death that is a result of respiratory failure due to RDS.

E.5.1.1

Timepoint(s) of evaluation of this end point

the first 72 hours of life

E.5.2

Secondary end point(s)

Secondary endpoints:
1. Incidence of respiratory failure or death due to RDS.
2. Incidence rate of BPD at 36 weeks PMA.
3. All-cause mortality
4. Incidence rate of survival without BPD at 36 weeks PMA.
5. Incidence of pulmonary air leak

Tertiary Endpoints:
The tertiary endpoints of this study include the evaluation of the following from the time of initiation of study treatment until study completion:
1. Incidence rates of common complications of prematurity other than air leak
2. Change from baseline in FiO2 and/or transcutaneous PCO2 over the first 72 hours of life

Safety Endpoints:
The following measures are to be documented in the electronic case report form (eCRF):
1.Survival (date and time of death, if applicable)
2.AEs, including adverse device effects (ADEs) and AEs of special interest including peridosing events, complications related to placement of bi-nasal prongs, and air leak
3.Concomitant medications
4.Use of respiratory support and supplemental O2 to include the following:
a)Need for endotracheal intubation and MV
b) Mode of respiratory support (including supplemental oxygen) and important parameters for that mode
5.Complications of prematurity
6.Physical examinations
7.Tolerability of lucinactant for inhalation
8.Incidence of air leak
9.Assessments of the following:
a)Vital signs
b)O2 saturation, as determined by pulse oximetry (SpO2)
c)Serum electrolyte measurements
d)Defecation
e)Chest radiography, prior to intubation

E.5.2.1

Timepoint(s) of evaluation of this end point

at 36 weeks PMA

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

Yes

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

Yes

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

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

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

nCPAP - Continuous positive airway pressure/nasal

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

Chile

Colombia

Hungary

Ireland

Netherlands

Poland

United States

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

Longer-Term Follow-Up - visit at 1-Year Corrected Age

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:

240

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:

240

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

Paediatric patients

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

140

F.4.2.2

In the whole clinical trial

240

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)

none

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2016-02-18

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-12-03

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2019-08-06

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IMP with orphan designation in the indication
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